Genetic Engineering in the Press by GEG
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June 20, 2022 6:32 AM
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Researchers create first comprehensive functional map of genes expressed in human cells

Researchers create first comprehensive functional map of genes expressed in human cells | Genetic Engineering in the Press by GEG | Scoop.it
The Human Genome Project was an ambitious initiative to sequence every piece of human DNA. The project drew together collaborators from research institutions around the world, including Whitehead Institute, and was finally completed in 2003.
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Jonathan Weissman, a member of the Whitehead Institute, and his colleagues have produced the first comprehensive functional map of genes expressed in human cells. The data from this project, published online on 9 June in Cell, associates each gene with its work in the cell. For this project, the researchers used the Perturb-seq method, which uses CRISPR/Cas9 genome editing to introduce genetic modifications into cells, and then uses single-cell RNA sequencing to capture information about the expressed RNAs resulting from a given genetic modification. Since RNAs control all aspects of cell behavior, this method can help decipher the many cellular effects of genetic modifications.  Using human blood cancer cell lines as well as non-cancerous retina-derived cells, the researchers performed a Perturb-seq on over 2.5 million cells and used the data to create a comprehensive map linking genotypes to phenotypes. Once completed, the researchers decided to use their new dataset and examine some biological questions. In the future, the researchers hope to use Perturb-seq on different cell types in addition to the cancer cell line they started with. They also hope to continue exploring their map of gene functions and hope that others will do the same. 

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July 4, 6:33 AM
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NUS researchers develop breakthrough gene delivery technology for immune cells

NUS researchers develop breakthrough gene delivery technology for immune cells | Genetic Engineering in the Press by GEG | Scoop.it
Researchers at the National University of Singapore (NUS) have developed a scalable, non-viral technology that efficiently delivers genetic material into human immune cells.
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CAR-T cell therapy is one of the most promising cancer treatment strategies to have emerged recently. This personalized approach has demonstrated its effectiveness, particularly in patients who have exhausted conventional options. However, it remains costly and logistically complex. One of the main obstacles to CAR-T manufacturing lies in the introduction of genetic material into immune cells. Methods currently used in the industry include viral vectors and mass electroporation. While viral approaches are effective, they raise concerns about safety, immunogenicity and random gene integration. What's more, mass electroporation, which relies on high-voltage electrical pulses, can stress and damage cells, reducing their therapeutic efficacy. A new NExT platform created by researchers overcomes these limitations. It works by connecting cells to a dense forest of nanopairs, microscopic hollow tubes less than a thousandth the width of a human hair. When a gentle electrical signal is applied, the nanopins open temporary pores in the cell membrane, allowing biomolecules such as mRNA or CRISPR/Cas9 complexes to penetrate directly into the cell cytoplasm.

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June 24, 10:01 AM
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Universal CAR T cell therapy shows promise for aggressive T cell cancers

Universal CAR T cell therapy shows promise for aggressive T cell cancers | Genetic Engineering in the Press by GEG | Scoop.it
A new type of immunotherapy that targets aggressive blood cancers shows promising results alongside manageable side effects, according to the results of an international phase 1/2 clinical trial led by researchers at Washington University School of Medicine in St. Louis.
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A new type of immunotherapy, called WU-CART-007, targeting aggressive blood cancers is showing promising results as well as manageable side effects, according to the results of an international phase 1/2 clinical trial conducted by researchers. Participants in the trial had been diagnosed with a rare cancer: T-cell acute lymphoblastic leukemia or T-cell lymphoblastic lymphoma, and had run out of treatment options as standard therapy had proved ineffective. In the process of its production, CRISPR deletes the T-cell receptor from donor cells, thereby considerably reducing the risk of graft-versus-host disease, in which donor T cells attack healthy tissue. Deletion of another key antigen also prevents CAR-T cells from attacking each other. After using CRISPR gene editing to modify CAR-T cells to prevent these harmful side effects, the cells are then modified to target a protein called CD7 on the surface of cancer T cells to destroy the cancer. The clinical trial evaluated the safety and efficacy of an innovative CAR-T cell-based immunotherapy, specifically designed to attack cancer T cells. Thanks to this new immunotherapy, most patients in the study who received the full dose of cells achieved complete remission of their cancer. 

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May 23, 6:22 AM
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Novel cancer immunotherapy approach could complement existing CAR-T treatments

Novel cancer immunotherapy approach could complement existing CAR-T treatments | Genetic Engineering in the Press by GEG | Scoop.it
Researchers in South Korea have proposed a novel cancer immunotherapy approach that could complement existing CAR-T (Chimeric Antigen Receptor T-cell) treatments.
BigField GEG Tech's insight:

Although highly effective against blood cancers such as leukemia, CAR-T therapy faces significant challenges in the treatment of solid tumors such as lung cancer. Macrophages, a type of immune cell, have the inherent ability to infiltrate solid tumors more effectively than T lymphocytes. This makes them a promising candidate for cancer treatment. However, existing macrophage-based therapies have limitations, not least the short duration of genetic modifications, which reduce therapeutic efficacy. A research team developed a series of innovative techniques to efficiently deliver synthetic CAR-encoding genes into macrophages without causing cell damage, by eliminating polybrene, optimizing the VSV-G (vesicular stomatitis virus G) protein, a key component facilitating viral entry into cells, and using the EF1α promoter, which enabled macrophages to maintain CAR gene expression for up to 20 days. CAR-M cells demonstrated potent antitumor effects. In co-culture with Nalm6 and Raji cells, CAR-M macrophages effectively engulfed and destroyed cancer cells, as observed by fluorescence microscopy. The research team plans to increase CAR-M production and further develop high-efficacy treatment protocols for clinical applications

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April 30, 12:28 PM
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Researchers develop a reverse genetics system for African swine fever virus

Researchers develop a reverse genetics system for African swine fever virus | Genetic Engineering in the Press by GEG | Scoop.it
Researchers from the J. Craig Venter Institute (JCVI), the Friedrich-Loeffler-Institut (FLI), and the International Livestock Research Institute (ILRI) have developed a reverse genetics system for African swine fever virus (ASFV).
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Researchers have developed a reverse genetics system for the African swine fever virus (ASFV). The scientists construct synthetic DNA, a laboratory-made version of the virus’s genetic material. Fragments of ASFV are then modified and assembled into complete genomes in yeast using its natural recombination mechanism. The modified genomes are subsequently transferred to E. coli, allowing them to be isolated in larger quantities. The synthetic DNA is then transfected (artificially introduced) into mammalian host cells, which are subsequently infected by a helper virus. This helper virus is an attenuated version of ASFV, modified using CRISPR/Cas9 technology—a powerful genetic editing tool capable of cutting DNA at precise locations. These alterations prevent the helper virus from replicating on its own. Despite this inhibition, the helper virus still provides the proteins and machinery necessary for the replication and assembly of the synthetic DNA into new viral particles. This process enables the production of live recombinant viruses containing the specific genetic modifications introduced into the synthetic DNA. The new system will help researchers develop vaccines and study the pathogenesis and biology of ASFV, a deadly and highly contagious viral disease affecting domestic and wild pigs.

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April 15, 6:20 AM
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New supercharged CAR-T cell therapy shows promise against resistant cancers

New supercharged CAR-T cell therapy shows promise against resistant cancers | Genetic Engineering in the Press by GEG | Scoop.it
Researchers at the University of Colorado Anschutz Medical Campus have successfully developed a supercharged iteration of CAR-T cell therapy that can enhance the effectiveness and longevity of the cells, particularly against cancer cells that are harder for prior CAR-T therapies to detect and fight.
BigField GEG Tech's insight:

Chimeric Antigen Receptor T-cell (CAR-T) therapy has revolutionized cancer treatment by engineering a patient's T-cells to target and eliminate malignant cells. However, certain cancers still evade detection, leading to treatment resistance and relapse. American researchers have recently introduced an innovative approach known as ALA-CART (adjunctive LAT-activating CAR-T cells), designed to overcome these challenges. In preclinical studies, ALA-CART demonstrated enhanced efficacy against acute lymphocytic leukemias that were unresponsive to traditional CAR-T therapies. By optimizing T-cell activation and targeting capabilities, this next-generation therapy not only improved cancer cell eradication but also showed potential in reducing associated side effects. These findings suggest that ALA-CART could offer a more robust and durable treatment option for patients with resistant cancers, marking a significant advancement in the field of immunotherapy.

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April 7, 5:08 AM
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David Liu Wins 2025 Breakthrough Prize for Base Editing and Prime Editing

David Liu Wins 2025 Breakthrough Prize for Base Editing and Prime Editing | Genetic Engineering in the Press by GEG | Scoop.it
New precision genome editing technologies are transforming the therapeutic landscape for patients with certain cancers and genetic diseases.
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This year’s Breakthrough Prize in Life Sciences has been awarded to David Liu, a biochemist at Harvard University, for the development of base editing and prime editing, two novel genome editing technologies. The Breakthrough Prize in Life Sciences honors researchers whose work has improved scientific understanding of living systems and contributed towards extending human life.

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March 18, 7:50 AM
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News: FDA Grants Dual Designations to Arbor's Gene Editing Therapy for Rare Kidney Disease

News: FDA Grants Dual Designations to Arbor's Gene Editing Therapy for Rare Kidney Disease | Genetic Engineering in the Press by GEG | Scoop.it
The FDA has granted orphan drug and rare pediatric disease designations to Arbor Biotechnologies' ABO-101, a gene-editing therapeutic candidate for primary hyperoxaluria type 1. The one-time treatment aims to reduce oxalate production in patients with this rare genetic kidney disease.
BigField GEG Tech's insight:

Primary hyperoxaluria (PH) is a group of rare, autosomal recessive metabolic diseases characterized by excessive oxalate production by the liver and subsequent accumulation of oxalate in the kidneys and other organs. Type I PH (PH1), the most common and severe subtype, is thought to affect 1 to 3 people per 1,000,000 in the general population. PH is incurable, and current therapeutic approaches focus on relieving symptoms and preventing the accumulation of oxalate in the kidneys and blood vessels. However, Arbor Biotechnologies has developed a treatment for PH1, called ABO-101, and announced in a recent press release that the FDA has granted both Orphan Drug Designation and Rare Pediatric Disease Designation to this treatment. ABO-101 is a novel investigational gene-editing therapy designed as a single liver-directed treatment that permanently deactivates the HAO1 gene to reduce PH1-associated oxalate production. The candidate uses a lipid nanoparticle delivery system (licensed from Acuitas Therapeutics) encapsulating messenger RNA expressing a CRISPR Cas12i2 type V nuclease and an optimized guide RNA targeting the HAO1 gene. 

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March 14, 11:41 AM
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Immune system discovery could revolutionize treatment for chronic disease and cancer

Immune system discovery could revolutionize treatment for chronic disease and cancer | Genetic Engineering in the Press by GEG | Scoop.it
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Chronic diseases such as cancer and chronic infections often leave the immune system in a state of exhaustion, where its front-line defenders, the T lymphocytes, lose their ability to function effectively. Research has identified a rare type of immune cell, called a T stem cell, which holds the key to maintaining robust, long-term immune responses. The study reveals that the stamina of these stem-type T lymphocytes is fuelled by a protein called ID3, expressed by a gene of the same name. These ID3+ T cells have a unique ability to self-renew and resist depletion, allowing them to maintain immune responses for much longer than other T cells that do not express ID3. The study also revealed that specific signals in the body could increase the number of ID3+ T cells, paving the way for improved treatments such as CAR T cell therapy. Although CAR T therapy has been transformative in treating certain cancers, its effectiveness can diminish over time due to T cell depletion.

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January 7, 6:20 AM
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Barcoding small extracellular vesicles with new CRISPR-based system

Barcoding small extracellular vesicles with new CRISPR-based system | Genetic Engineering in the Press by GEG | Scoop.it
Cell-to-cell communication through nanosized particles, working as messengers and carriers, can now be analyzed in a whole new way, thanks to a new method involving CRISPR gene-editing technology.
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Researchers at the University of Tokyo have developed a groundbreaking CRISPR-based barcoding system to study small extracellular vesicles (sEVs), nanosized particles crucial in intercellular communication and disease progression. The system uses CRISPR gene-editing technology to introduce RNA barcodes into sEVs, allowing thousands of genes to be analyzed simultaneously in a single pooled experiment. CIBER (CRISPR-assisted individually barcoded extracellular vesicle-based release regulator) offers detailed insights into sEV subpopulations, enabling more efficient and comprehensive studies than conventional methods. This innovation opens new pathways for sEV-based diagnostics and therapeutic applications. 

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December 4, 2024 6:33 AM
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Modulating CD39 expression to enhance CAR-T cell therapy against hepatocellular carcinoma

Modulating CD39 expression to enhance CAR-T cell therapy against hepatocellular carcinoma | Genetic Engineering in the Press by GEG | Scoop.it

In the quest to enhance the efficacy of chimeric antigen receptor T (CAR-T) cell therapy against hepatocellular carcinoma (HCC), a recent study has identified a pivotal role for CD39 expression in modulating the function of CAR-T cells.

BigField GEG Tech's insight:

In the quest to improve the efficacy of CAR-T cell therapy against hepatocellular carcinoma (HCC), a recent study has identified a pivotal role for CD39 expression in modulating CAR-T cell function. The research, conducted by a team of scientists, investigated the impact of CD39 modulation on CAR-T cells, hypothesising that optimal levels of CD39 could increase the therapeutic potential of these cells against HCC. The study began with the isolation and culture of primary human T lymphocytes, transduced with a lentiviral vector encoding the CAR CD39 construct. The study also used a combination of in vitro and in vivo experiments, including a mouse model of subcutaneous HCC, to demonstrate that CAR-T cells with moderate levels of CD39 expression displayed superior antitumour activity to those with high or low levels of CD39. In addition, the study explored the use of mdivi-1, a small molecule inhibitor, to modulate CD39 expression, revealing a synergistic effect when combined with CD39 suppression that significantly improved the antitumour response. 

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November 25, 2024 9:59 AM
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New CAR T cells offer controlled treatment for solid tumors

New CAR T cells offer controlled treatment for solid tumors | Genetic Engineering in the Press by GEG | Scoop.it
Ludwig Cancer Research scientists have devised new types of chimeric antigen-receptor (CAR) T cells-;a type of cancer immunotherapy-;that can be switched on to varying degrees of intensity and then switched off on demand with existing drugs.
BigField GEG Tech's insight:

When the CAR binds its antigen to a cancer cell, its signalling modules are activated, triggering the T cells' innate cytotoxic armamentarium to destroy the tumour cells. The problem is that many solid tumour antigens are also found on healthy cells, increasing the risk of so-called ‘off-target, off-tumour’ effects. Researchers have developed new types of CAR-T cells, a type of cancer immunotherapy that can be activated at different intensity levels and then deactivated on demand with existing drugs. To enable control of CAR activity, researchers have separated the antigen-sensing part (the antibody fragment) and the activation domain (CD3-ζ) into two distinct chains, the ‘receptor chain’ and the ‘signalling chain’. They have also included an additional module capable of dimerising the two chains upon application of a cancer drug called venetoclax. However, CAR-T cells must also be rapidly deactivated if they are dangerous to patients. To this end, the researchers added a drug component to the CD3-ζ signalling chain that responds to another approved cancer drug, lenalidomide. The researchers show that iON/OFF CAR all-in-one T cells (iONØ-CAR) can be activated by venetoclax and rapidly deactivated - in 4 to 6 hours - by lenalidomide.

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November 15, 2024 10:07 AM
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This scientist treated her own cancer with viruses she grew in the lab - Nature

This scientist treated her own cancer with viruses she grew in the lab - Nature | Genetic Engineering in the Press by GEG | Scoop.it

Virologist Beata Halassy says self-treatment worked and was a positive experience — but researchers warn that it is not something others should try.

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Virologist Beata Halassy says self-treatment worked and was a positive experience — but researchers warn that it is not something others should try. 

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October 14, 2024 8:28 AM
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News: CRISPR Fixes Multiple Dystrophin Duplications in DMD Patient Cells

News: CRISPR Fixes Multiple Dystrophin Duplications in DMD Patient Cells | Genetic Engineering in the Press by GEG | Scoop.it
Researchers in France report the correction of three duplications in the dystrophin gene in cells from Duchenne muscular dystrophy patients, using CRISPR-Cas9 gene editing and a single guide RNA. The findings highlight the potential of using CRISPR-Cas9 to correct DMD duplications in exons that are not addressed by any of the fou
BigField GEG Tech's insight:

Duchenne muscular dystrophy (DMD) is a rare, incurable muscular disease affecting around 1 in every 3,500 to 5,000 male births worldwide. The disease follows an X-linked pattern of inheritance, and exonic duplications of the gene coding for DMD dystrophin are frequently observed in DMD patients. Dystrophin is a cytoplasmic protein that plays a mechanical role in muscle. In a recent study, French researchers used the CRISPR-Cas9 gene-editing technique to target intronic regions of the DMD gene. Their aim was to delete certain duplicated regions in patients' immortalized myogenic (muscle progenitor) cells, in particular duplications of exon 2, exons 2 to 9 or exons 8 to 9, which are known hotspots for mutations in DMD patients.They confirmed restoration of the DMD open reading frame and rescued dystrophin expression by Western blotting and mytotube immunostaining after CRISPR-based deletion of the target duplications. RNA sequencing suggested gene rescue in dystrophin-related pathways. Off-target analysis based on predicted nearby off-targets revealed no significant unintended genetic changes at these loci.

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July 4, 3:29 AM
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AbbVie to buy Capstan for up to $2.1 billion in immunology push

AbbVie to buy Capstan for up to $2.1 billion in immunology push | Genetic Engineering in the Press by GEG | Scoop.it
AbbVie said on Monday it would acquire privately held cell therapy developer Capstan Therapeutics in a deal worth up to $2.1 billion, expanding its product pipeline with experimental treatments for autoimmune diseases.
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AbbVie said on Monday it would acquire privately held cell therapy developer Capstan Therapeutics in a deal worth up to $2.1 billion, expanding its product pipeline with experimental treatments for autoimmune diseases.

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May 26, 6:57 AM
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lncRNAs shape the tumor microenvironment and drive cancer development

lncRNAs shape the tumor microenvironment and drive cancer development | Genetic Engineering in the Press by GEG | Scoop.it
The tumor microenvironment (TME) is a complex and dynamic network consisting of tumor cells, immune cells, stromal cells, extracellular matrix (ECM), cytokines, and growth factors, all interacting to influence tumorigenesis, progression, and metastasis.
BigField GEG Tech's insight:

The tumor microenvironment (TME) is a complex and dynamic network composed of tumor cells, immune cells, stromal cells, extracellular matrix (ECM), cytokines and growth factors, all of which interact to influence tumorigenesis, progression and metastasis. Long non-coding RNAs (lncRNAs), a class of non-coding RNAs over 200 nucleotides in length, have recently attracted considerable interest for their role in regulating gene expression within the MCT. They contribute to crucial processes such as immune evasion, angiogenesis, metabolic reprogramming and cancer stem cell maintenance, and their influence extends to the transcriptional, post-transcriptional and epigenetic levels. Targeting lncRNAs represents a promising therapeutic strategy for disrupting tumor-stroma interactions and improving the efficacy of current treatments. As research continues to reveal the complex roles of lncRNAs in cancer biology, these molecules have the potential to revolutionize cancer diagnosis and treatment, offering new hope to patients with advanced, treatment-resistant cancers.

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May 14, 11:43 AM
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Researchers develop tool to enhance NK cells against cancer

Researchers develop tool to enhance NK cells against cancer | Genetic Engineering in the Press by GEG | Scoop.it
A team of researchers from the Hospital del Mar Research Institute, the Universitat Autònoma de Barcelona and the Pompeu Fabra University has developed a new tool that allows modifying these NK cells to make them immune to the tumor’s defense mechanism.
BigField GEG Tech's insight:

One of the functions of Natural Killer (NK) cells is to detect and eliminate cancer cells. However, in some cases they are unable to overcome the tumour's defence mechanism, and cancer develops. To date, NK lymphocyte-based treatments have been effective in haematological tumours, but have not achieved the same level of efficacy in solid tumours. A study published in Nature Immunology proposes a new approach for strengthening NK cells in their fight against tumour cells. Using CRISPR-Cas 9, the researchers deactivated a specific gene, SMAD4, involved in TGF-β and activin A signalling in preclinical models of HER2-positive breast cancer tumour cells and metastatic colorectal cancer cells. The modified NK cells were able to overcome the negative effects of TGF-β and activin A.

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April 16, 3:58 AM
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Are Allogeneic CAR-Ts the Future of LBCL Cancer Treatment?

Are Allogeneic CAR-Ts the Future of LBCL Cancer Treatment? | Genetic Engineering in the Press by GEG | Scoop.it
Allogene Therapeutics recently released exciting results from the Phase 1 ALPHA/ALPHA2 trials of the Allogeneic CAR T 'Cemacabtagene Ansegedleucel/ALLO-501' in Relapsed/Refractory Large B-Cell Lymphoma.
BigField GEG Tech's insight:

Recent developments in allogeneic CAR-T cell therapy have shown promise in treating relapsed or refractory large B-cell lymphoma (LBCL). Californian biotech Allogene Therapeutics reported encouraging results from their Phase 1 ALPHA and ALPHA2 trials, evaluating the efficacy of ALLO-501, an allogeneic CAR-T product, in LBCL patients. The trials demonstrated complete response rates of 67% and 58%, respectively, with a median duration of response of 23.1 months among responders. Notably, patients with minimal disease burden at treatment initiation exhibited particularly favorable outcomes. These findings suggest that allogeneic CAR-T therapies could serve as effective "off-the-shelf" treatments, offering a more accessible and timely alternative to autologous CAR-T therapies, which require individualized cell harvesting and processing. Pitfalls however include accepting the “non-self” and graft versus-host disease (GvHD). An ongoing third trial aims to further assess the potential of this approach to redefine standard care practices in oncology

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April 8, 6:34 AM
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The Interface of Gene Editing with Regenerative Medicine - ScienceDirect

The Interface of Gene Editing with Regenerative Medicine - ScienceDirect | Genetic Engineering in the Press by GEG | Scoop.it
The potential of regenerative medicine in the clinical space is vast, given its ability to repair and replace damaged tissues, restore lost functions …
BigField GEG Tech's insight:

Recent developments in CRISPR-Cas9 gene-editing technology have significantly advanced the field of regenerative medicine. CRISPR-Cas 9 offers precise and efficient methods for tissue repair and the treatment of genetic disorders. It enables targeted genome modifications, including gene knock-ins, knockouts, and base conversions, facilitating the correction of mutations responsible for diseases such as cystic fibrosis, sickle cell disease, and osteogenesis imperfecta. Beyond genetic corrections, CRISPR is instrumental in reprogramming somatic cells into induced pluripotent stem cells (iPSCs), which can then be differentiated into specific cell types for therapeutic applications. It is also a key research tool facilitating genetic screening and disease models creation. While challenges remain, especially with safe delivery and minimizing off-target effects, the potential is undeniably vast. These advancements underscore CRISPR's pivotal role in enhancing tissue repair processes and developing innovative treatments for previously intractable conditions. 

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March 20, 11:51 AM
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New study confirms CAR T cell therapy does not cause secondary cancers

New study confirms CAR T cell therapy does not cause secondary cancers | Genetic Engineering in the Press by GEG | Scoop.it
Within a large group of more than 700 patients treated with CAR T cell therapy, researchers found no evidence that the therapy itself caused any type of secondary cancer in the modified T cells, according to new analysis reported today in Nature Medicine from the Perelman School of Medicine at the University of Pennsylvania and Penn Medicine's Abramson Cancer Center.
BigField GEG Tech's insight:

CAR T cell therapy is a personalized form of immunotherapy that uses a deactivated virus to program an individual's T cells to target and kill their cancer cells. Since the first CAR T cell therapy was approved in 2017, more than 30,000 patients with blood cancers have been treated. Some of the first patients treated in clinical trials have experienced durable remissions of a decade or more. In late 2023, the FDA announced that it was investigating several reported cases of secondary T-cell malignancies in patients who had previously received CAR T cell therapy products. In 2024, the FDA also began requiring drug manufacturers to add a safety warning to the label of CAR T cell products. As a result, researchers analysed samples from 783 adult and paediatric patients in Philadelphia who had been treated with CAR T cell therapy in clinical trials and found 18 cases of secondary cancers. None of the 18 cases showed evidence that they were caused by insertional mutagenesis. The researchers attributed the rarity of secondary cancers to immune system suppression due to previous anti-cancer treatments.

 

 

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March 17, 11:19 AM
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New gene modification enhances CAR-T cell effectiveness against cancer

New gene modification enhances CAR-T cell effectiveness against cancer | Genetic Engineering in the Press by GEG | Scoop.it
Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying a specific gene improves the ability of immune cells to combat cancer for an extended period, potentially reducing the chance of cancer recurring.
BigField GEG Tech's insight:

Although clinicians have successfully used CAR-T cells in cancer, many patients experience a cancer relapse caused by the hostile environment created by the cancer cells. After repeated encounters with cancer cells, CAR-T cells lose their ability to effectively divide and attack the tumour. Therefore, a research team used CRISPR screening to identify potential genes that could improve CAR-T cell therapy. The researchers found that knocking out the CUL5 gene improved the growth and longevity of CAR-T cells, even after repeated exposure to cancer cells. The gene of interest, CUL5, is involved in the degradation of specific proteins inside cells. When this gene is inactive, a cell signalling pathway known as the JAK-STAT signalling pathway becomes more sustained. This pathway sends signals encouraging T lymphocytes to grow and multiply, making them more effective at fighting cancer. Therefore, a new way of partially reducing CUL5 activity has been developed by using a virus to deliver genetic material to CAR-T cells.

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February 3, 10:00 AM
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New therapeutic target could boost CAR T-cell therapy success in solid tumors

New therapeutic target could boost CAR T-cell therapy success in solid tumors | Genetic Engineering in the Press by GEG | Scoop.it
Russell W. Jenkins, MD, PhD, a physician investigator in the Krantz Family Center for Cancer Research at the Mass General Cancer Center and an assistant professor of Medicine at Harvard Medical School, is senior author of a new study in Cancer Immunology Research, "TBK1 is Identified as a Therapeutic Strategy to Enhance CAR T-Cell Efficacy Using Patient-Derived Organotypic Tumor Spheroids".
BigField GEG Tech's insight:

CAR-T therapy treatments have proven effective in patients with blood tumors, however, their efficacy in solid tumors has been limited due to a variety of factors, including the tumor microenvironment. For this reason, researchers used a 3D microfluidic model made from patient-derived organotypic tumor spheroids (PDOTS) to study the mechanisms of treatment resistance of CAR-T cells designed in particular to target B7-H3, a common antigen in solid tumor cancers. By inhibiting the function of TBK1, a gene previously associated with immune evasion, they were able to restore CAR-T cell activity, prevent dysfunction and increase T cell proliferation. The researchers also found that inhibiting or suppressing TBK1 made cancer cells more sensitive to immune cell targeting and killing. The results therefore suggest that targeting TBK1 could reduce treatment resistance and improve CAR T efficacy in solid tumor cells expressing B7-H3, and also demonstrate the feasibility and utility of using PDOTS to study tumor-immune cell interactions. 

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December 13, 2024 6:29 AM
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Eleven clinical trials that will shape medicine in 2025 | Nature Medicine

Eleven clinical trials that will shape medicine in 2025 | Nature Medicine | Genetic Engineering in the Press by GEG | Scoop.it

Nature Medicine asks leading researchers to name their top clinical trial for 2025, from gene therapies for prion disease and sickle-cell disease to digital tools for cancer and mental health.

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Nature Medicine asks leading researchers to name their top clinical trial for 2025, from gene therapies for prion disease and sickle-cell disease to digital tools for cancer and mental health.

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December 2, 2024 7:05 AM
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News: CRISPR Plus Drug Treatment Makes Neuroblastoma Responsive to Immunotherapy

News: CRISPR Plus Drug Treatment Makes Neuroblastoma Responsive to Immunotherapy | Genetic Engineering in the Press by GEG | Scoop.it
Researchers in Italy and Australia report a triple combination strategy involving CRISPR that boosts the response of neuroblastoma to immunotherapy.
BigField GEG Tech's insight:

Neuroblastoma (NB) is a fatal childhood cancer that does not respond well to checkpoint immunotherapy due to low immunogenicity caused by low MHC class I expression and low neoantigen load. Researchers found that CRISPR-mediated silencing of the ERAP 1 (Endoplasmic Reticulum AminoPeptidase 1) gene in combination with entinostat, a histone deacetylase inhibitor, increased the immunogenicity of NB cells, making them more sensitive to PD-1 treatment. ERAP is an enzyme that cleaves peptides before loading them onto MHC class I molecules, and its inhibition leads to the generation of new antigens capable of inducing robust anti-tumour immune responses. The team observed that although none of the strategies was compelling, a triple combination of ERAP 1 silencing + Entinostat + PD-1 blockade significantly improved toxicity-free survival rates in a mouse model of NB. The approach is promising because it could help overcome the low immunogenicity of NB, making it more sensitive to immunotherapy.

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November 22, 2024 6:49 AM
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Overcoming CRISPR's side effects for therapeutic applications

Overcoming CRISPR's side effects for therapeutic applications | Genetic Engineering in the Press by GEG | Scoop.it
CRISPR is a revolutionary tool that allows scientists to precisely modify the genome and gene expression of cells in any organism. It's a reagent-;a substance that facilitates a reaction-;that combines an enzyme with a programmable RNA capable of locating specific genetic sequences. Once guided to the correct spot, the enzyme acts like a pair of scissors, cutting, replacing, or deleting sequences of DNA.
BigField GEG Tech's insight:

The CRISPR tool is very powerful, but it has side effects. By modifying one gene, CRISPR can activate or deactivate many associated genes, leading to unexpected results. To combat these side effects, a team of researchers uses computer modeling and deep learning to predict the broader impacts of CRISPR gene modifications on the genome. The model allows the team to simulate the effects of modifying a single gene on the whole genome, enabling them to predict and avoid unforeseen consequences. It also contributes to the evaluation and identification of new genetic targets. Their approach has far-reaching implications in various fields, including cancer treatment and musculoskeletal applications. For example, the team has identified candidate genes that can enhance the differentiation of induced pluripotent stem cells into more effective cancer-fighting cells. Furthermore, CRISPR must penetrate the cell's nucleus using nanoparticles to function. However, the researchers realized that the nanoparticles can negatively affect the cells. They, therefore, used various computer modeling techniques to predict how these mechanisms affect the ability of stem cells to differentiate and survive.

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October 16, 2024 4:25 AM
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VitelloTag approach offers new solution for gene editing challenges in egg cells

VitelloTag approach offers new solution for gene editing challenges in egg cells | Genetic Engineering in the Press by GEG | Scoop.it
A new approach for delivering miniature research tools into the interior of egg cells and embryos has been developed at the Marine Biological Laboratory (MBL), resolving a major bottleneck to using the gene-editing tool CRISPR-Cas9 in many research organisms.
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Scientists use a long, thin glass needle to inject eggs or embryos, a technique known as microinjection, which requires a great deal of time and expertise. However, a new delivery approach has been inspired by an egg yolk protein found in most animals, called vitellogenin, which provides an energy source for the growing egg. Vitellogenin is a large protein, but the team isolated the small part that binds to the receptor on the surface of the egg. This is a very small tag (around 10 amino acids) to which various fillers, such as Cas9, can be added. The team has successfully used VitelloTag on two distantly related species: the starfish (Patiria miniata) and the acorn worm (Saccoglossus kowalevskii). However, microinjection remains the method of choice for delivering CRISPR-Cas9 to many organisms. Penetration (the percentage of cells that successfully absorb the CRISPR load) can reach 90% with microinjection, while with VitelloTag, the team achieved around 30% penetration in starfish and acorn worms. 

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