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Ide-Cel Demonstrates Durable Responses in Relapsed/Refractory Multiple Myeloma

Ide-Cel Demonstrates Durable Responses in Relapsed/Refractory Multiple Myeloma | Genetic Engineering - GEG Tech top picks | Scoop.it
The CAR T-cell therapy idecabtagene vicleucel continues to demonstrate improved survival among heavily pretreated patients with relapsed/refractory multiple myeloma.
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The CAR T-cell therapy idecabtagene vicleucel (ide-cel; Abecma) continues to demonstrate improved survival in heavily treated patients with relapsed/refractory multiple myeloma, according to updated results from the phase 2 KarMMa trial (NCT03361748) presented at the

2021 ASCO Annual Meeting. During the trial, 140 patients with at least three prior lines of treatment for multiple myeloma who were refractory to their last treatment regimen were enrolled. However, only 128 patients received an ide-cel infusion. The overall response rate was 73% in the overall population, with 33% complete response, 20% very good partial response and 20% partial response. The median time to first response was 1 month, with a median time to complete response of 2.8 months. In addition, the rapid response rate did not vary with the number of prior treatments received. The safety profile of ide-cel was consistent with long-term follow-up, with similar rates of infections and secondary primary malignancies, and no unexpected gene therapy-related toxicities were observed.

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First CAR-T therapy to target BCMA gets FDA nod - Nature

First CAR-T therapy to target BCMA gets FDA nod - Nature | Genetic Engineering - GEG Tech top picks | Scoop.it
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The first CAR-T immunotherapy directed against BCMA (idecabtagene vicleucel) for patients with relapsed or refractory multiple myeloma developed by partners Bristol Myers Squibb and bluebird bio was approved by the U.S. Food and Drug Administration on March 26, 2021.

This therapy recognizes and binds BCMA (B cell maturation antigen) which is a cell surface protein that is expressed on a majority of cancerous B cells in multiple myeloma. The approval is based on the KarMMa trial of 127 patients, 100 of whom received the personalized treatment with genetically engineered autologous T cells. Their overall response rate was 72%, and 28% achieved a strict complete response. For Bristol Myers Squibb, this is the second approval for a CAR-T cell therapy. The first was for a therapy directed against CD19.

Bristol Myers Squibb and bluebird will jointly market their immunotherapy at a list price of $419,500.

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News: Simplest Possible Modification Rescues Dystrophin Expression in Duchenne

News: Simplest Possible Modification Rescues Dystrophin Expression in Duchenne | Genetic Engineering - GEG Tech top picks | Scoop.it
Editing a single nucleotide in the largest human gene is sufficient to restore dystrophin production and myocyte function in Duchenne muscular dystrophy. The new research uses base editing and prime editing that only nicks a single strand of DNA and reduces the risk of damaging genetic changes.
BigField GEG Tech's insight:

To restore dystrophin production and myocyte function in patients with Duchenne muscular dystrophy (DMD), several CRISPR strategies have been tried to correct some of the hundreds of thousands of documented mutations in the dystrophin gene (DMD). Among these strategies, researchers at the University of Texas Southwestern Medical Center in Dallas, Texas, have developed a novel CRISPR gene editing strategy for DMD therapy. This was tested in animal and human DMD models where the researchers deleted exon 51 in the DMD gene which disrupted the dystrophin reading frame and generated a premature stop codon in exon 52. The researchers were able to restore the reading frame with both basic and main editing by introducing exon skipping and reframing, respectively, through their "single-swap" ABE strategy, i.e., through editing a single letter in the splice acceptor or donor site can cause exon skipping. The researchers targeted the splice acceptor or splice donor site of exon 50 or 52 and the single nucleotide modification restored dystrophin expression in human cardiomyocytes and contractile function was normalized using master editing. However, one challenge is the high doses required to deliver expression throughout the body.

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Parallel Genome Editing in Microscopic Worms Maps Regulatory Genomic Elements to Physiology

Parallel Genome Editing in Microscopic Worms Maps Regulatory Genomic Elements to Physiology | Genetic Engineering - GEG Tech top picks | Scoop.it
A group of systems biologists in Berlin have developed parallel genome editing in tiny worms to produce diverse indel mutations in regulatory elements in genomic DNA and a powerful software package, crispr-DART, to analyze the indel mutations following targeted DNA sequencing. Using this new approach, they directly map gene regulatory genotypes to physical and physiological attributes in the worm.
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The human genome is made up of 40% of regulatory elements that control gene expression. Understanding the function of these regulatory regions is very important for understanding the cause of certain diseases. The study of these regions is complicated because they must be in the context of their genomic and tissue environment and their developmental timeline. To overcome this, researchers at the Max Delbrück Center for Molecular Medicine in Berlin introduced various large-scale mutations using CRISPR-Cas9 in the form of deletions or insertions into the genomes of thousands of C. elegans worms and then monitored the physiological effect of these mutations. In addition, one of the team's bioinformatics researchers developed sequencing software called CRISPR- Downstream Analysis and Reporting Tool (DART), to analyze the generated data. One of their results was the identification of the function of two let-7 microRNA binding sites that work independently in the downstream regulatory region of a gene called lin-41. They were able to show that if one of the two sites were intact, the worms grew normally, otherwise gene expression was incorrect and the worms grew poorly and died.

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Researchers devise more efficient, enduring CAR gene therapy to combat HIV - UCLA

Researchers devise more efficient, enduring CAR gene therapy to combat HIV - UCLA | Genetic Engineering - GEG Tech top picks | Scoop.it
Research brief: A UCLA team has demonstrated that altering a key molecule used in the therapy yields superior and longer-lasting results in mouse models.
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A new approach to CAR T cells therapy developed by a UCLA research team allows for an effective and long-lasting response to HIV, unlike current applications of such therapies that cannot provide lasting immunity, i.e., they cannot destroy malignant or infected cells that appear months or years after treatment. Previously, researchers had created a therapy based on CAR T cells containing part of the CD4 molecule because HIV binds to CD4 molecules in order to infect cells in the body. HIV binds to the CD4 molecule of the CAR T cell, which activates it and the cell kills HIV. However, two domains of CD4 molecules still allowed HIV to infect cells. So, the researchers removed these domains and added another that makes the cells resistant to infection and allows a more effective and durable cellular response against HIV than the former therapy. This new CAR T cells therapy with a truncated form of the CD4 molecule works like a vaccine. It stimulates the patient's immune system before HIV itself induces a response. In addition, this therapy induces the production of a large number of memory T cells and could influence the field of immunotherapy focused on engineering T cells with CAR molecules.

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Scientists launch clinical trial of CRISPR gene correction therapy in patients with sickle cell disease

Scientists launch clinical trial of CRISPR gene correction therapy in patients with sickle cell disease | Genetic Engineering - GEG Tech top picks | Scoop.it
Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient's own blood-forming stem cells.
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Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin. This disease affects millions of people worldwide. It reduces the ability of red blood cells to carry oxygen efficiently and progresses to chronic vascular disease. The disease is caused by a single letter mutation in human DNA at a gene in beta-globulin. To combat this disease, researchers at UC San Francisco, UC Berkeley and UCLA have developed a CRISPR-based gene correction therapy, named CRISPR_SCD001, for patients with sickle cell disease. This therapy is based on the use of blood stem cells taken from patients that are modified with a CRISPR-Cas9 nuclease introduced into the cells by electroporation in order to stimulate repair of the sickle cell mutation that causes the patients to form deformed red blood cells. The modified stem cells are then reintroduced into the patient. This is the first-time researchers will attempt to correct the beta-globulin gene in patients' own cells using non-viral delivered CRISPR tools. Their therapy has received approval from the U.S. Food and Drug Administration for an initial human clinical trial.

The study will last 4 years and will include 6 adults and 3 adolescents with severe sickle cell disease.

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CAR T-cell therapy triples expected length of remission for relapsed multiple myeloma patients

CAR T-cell therapy triples expected length of remission for relapsed multiple myeloma patients | Genetic Engineering - GEG Tech top picks | Scoop.it
A new type of CAR T-cell therapy more than triples the expected length of remission for multiple myeloma patients who have relapsed several times, according to an international clinical trial with UT Southwestern as the lead enrolling site.
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Multiple myeloma, the second most common blood cancer, is a cancer of plasma cells, an important white blood cell in the immune system. The disease's attack on the bone marrow puts patients at risk of life-threatening infections. So far, most treatments induce responses in only a third of patients, and complete remissions are rare. However, a new therapy based on CAR-T cells, called idecabtagene vicleucel, has been developed: patients' T cells have been modified to target a molecule called B-cell maturation antigen, which is only found in plasma cells and myeloma cells. This therapy could triple the expected duration of remission for multiple myeloma patients who have repeatedly relapsed. The results of the trial, published recently in the New England Journal of Medicine, were significantly better than those seen with other available therapies for patients with highly relapsed and refractory myeloma who had already received the three main classes of treatment. Nearly three-quarters of patients had at least a partial response to therapy. About one-third achieved complete remission, with all traces of cancer gone. 

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News: An Arms Race Can Make the CRISPR Toolkit Even Better

News: An Arms Race Can Make the CRISPR Toolkit Even Better | Genetic Engineering - GEG Tech top picks | Scoop.it
A constant battle between prokaryotes and phages has shaped how CRISPR is controlled and regulated. Haridha Shivram from Jennifer Doudna’s lab is working to understand these mechanisms, and in a recent review, he describes how this knowledge might enable us to build a better genome-editing tool.
BigField GEG Tech's insight:

About half of all bacteria studied and 90% of archaea use CRISPR-Cas systems to defend themselves against phages. In addition, prokaryotes have developed multiple mechanisms to control the expression of the CRISPR-Cas gene so that the defense can be stopped or activated as needed. However, phages retaliate and attempt to counter the defense via a diverse set of anti-CRISPR proteins (Acrs) that inhibit CRISPR-Cas activity and thus help the phage to successfully attack bacteria. Acrs have been experimentally exploited to reduce off-target edition by reducing the time available for Cas nuclease activity and increase tissue-specific edition by controlling their presence in various tissues. Understanding mechanically how CRISPR is regulated could lead to the improvement of CRISPR as a genome editing tool. Haridha Shivram, post-doctoral fellow at the University of California at Berkeley, has the research objective to discover new regulators of CRISPR-Case systems. The most fascinating aspect for him is how the arms race between the host and its invasive mobile genetic elements can lead to unique molecular innovations that can both help boost immunity but also reveal their hidden vulnerabilities. These evolving strategies can help make the CRISPR toolbox even better.

 

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Modified version of CAR T-cell therapy shows promise in targeting neuroblastoma

Modified version of CAR T-cell therapy shows promise in targeting neuroblastoma | Genetic Engineering - GEG Tech top picks | Scoop.it
Chimeric Antigen Receptor T-cell therapy--CAR T--has revolutionized leukemia treatment. Unfortunately, the therapy has not been effective for treating solid tumors including childhood cancers such as neuroblastoma.
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The development of CAR T cells has facilitated the treatment of blood tumours. Furthermore, this therapy is not effective against solid tumours such as neuroblastoma and has even revealed toxic effects which are due to the fact that most of the antigens that cancerous tissue has on its surface are also found in healthy tissue. However, a group of scientists at Los Angeles Children's Hospital has developed a modified version of CAR T cells that looks promising for targeting neuroblastoma based on the pre-clinical phases. Their study was published in Nature Communications. The researchers used a new CAR T technology called Synthetic Notch (synNotch). SynNotch CAR T cells have a unique property. The special synNotch protein is designed to recognize the GD2 antigen. When it does, this protein instructs the cell to activate its CAR T properties, allowing it to recognize a second antigen: B7H3. By following these specific instructions, cells can only kill cells with both antigens and therefore mostly cancer cells. This triggering property minimizes toxicity because healthy cells will sometimes have low levels of one of the antigens but never both.  

 

https://www.nature.com/articles/s41467-020-20785-x

 

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News: Gene-Editing IND and Pre-clinical Update

News: Gene-Editing IND and Pre-clinical Update | Genetic Engineering - GEG Tech top picks | Scoop.it
Update on Allogene Therapeutics off-the-shelf CAR-T cancer therapies. This week’s gene-editing update looks at an investigational new drug (IND) programme and a pre-clinical programme for gene-edited CAR T-cell therapies for renal cell carcinoma, haematological cancers, and multiple myeloma.
BigField GEG Tech's insight:

Allogene Therapeutics develops allogeneic CAR T cell-based therapies for a range of hematological and solid cancers. Two candidates are being developed using Allogene's exclusive Allo CAR T platform : 

  • - ALLO-316 is an AlloCAR T ™ anti-CD70 candidate in development for the treatment of renal cell carcinoma as well as several haematological cancers that express the CD70 cell surface antigen. CD52 is also disrupted in order to make CAR T cells resistant to this treatment. Allogene announced that the FDA has approved a phase 1 clinical trial in patients with advanced or metastatic renal cell carcinoma. This is the company's first clinical trial in solid tumours.  
  • - ALLO-605 is a TurboCAR ™, under development for multiple myeloma, targeting B cell maturation antigen (BCMA), a cell surface protein universally expressed on malignant plasma cells. The company presented preclinical data that demonstrated improved cytokine secretion, polyfunctionality, improved in vitro serial killing activity, and improved anti-tumor activity and survival compared to CAR T cells targeting BCMA in a mouse model aggressive for multiple myeloma. Allogene revealed that it expects to file our first Investigational New Drug application for its new TurboCAR technology ™ in the first half of 2021. 
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Penn study opens the door to let CAR T cells reach the tumor microenvironment

Penn study opens the door to let CAR T cells reach the tumor microenvironment | Genetic Engineering - GEG Tech top picks | Scoop.it
Targeting PAK4 to reprogram the vascular microenvironment and improve CAR-T immunotherapy for glioblastoma
BigField GEG Tech's insight:

The network of blood vessels in the tumour microenvironment remains one of the most difficult blockages for cell therapies to penetrate and treat solid tumours. However, a new study published in Nature Cancer explains that researchers at Penn Medicine found that combining CAR T cell therapy with a PAK4 inhibitor drug allowed modified cells to work their way through and attack the tumour, leading to significantly improved survival in mice. The genetic reprogramming of tumour endothelial cells lining the walls of blood vessels is caused by an enzyme known as PAK4. Penn's team discovered that PAK4 inhibition reduces abnormal tumour vascularization and improves T cell infiltration and CAR T cell immunotherapies in mouse models of glioblastoma. An experiment with T-RCA cell therapy led by EGFRvIII and a PAK4 inhibitor showed a nearly 80 percent reduction in tumour growth compared to mice that received CAR T cell therapy only five days after infusion. The targeting of PAK4 may therefore provide a unique opportunity to recondition the tumour microenvironment and improve T-cell-based cancer immunotherapy for solid tumours.   

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Novel Dual CAR T Cell immunotherapy Holds Promise for Targeting The HIV Reservoir – PR News

Novel Dual CAR T Cell immunotherapy Holds Promise for Targeting The HIV Reservoir – PR News | Genetic Engineering - GEG Tech top picks | Scoop.it

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BigField GEG Tech's insight:

A recent study published in the journal Nature Medicine, led by researchers James Riley, PhD, a professor of Microbiology at the Perelman School of Medicine at the University of Pennsylvania, and Todd Allen, PhD, a professor of Medicine at Harvard Medical School and Group Leader at the Ragon Institute of MGH, MIT and Harvard, describes a new Dual CAR T cell immunotherapy that can help fight HIV infection.

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Chlorotoxin-directed CAR T cells for specific and effective targeting of glioblastoma - Science Translational Medicine

Chlorotoxin-directed CAR T cells for specific and effective targeting of glioblastoma - Science Translational Medicine | Genetic Engineering - GEG Tech top picks | Scoop.it
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Chlorotoxin derived from scorpion venom has previously been shown to bind glioblastoma cells. Wang et al. designed a chimeric antigen receptor (CAR) based on chlorotoxin to surmount limitations of other glioblastoma-targeted CARs that have not been able to overcome tumor heterogeneity and antigen escape. They demonstrated that chlorotoxin binding captures a broader array of primary tumors than staining for previously identified antigenic targets. Chlorotoxin-directed CAR T cells were safe in mice and induced regression of orthotopic glioblastoma xenografts with no evidence of antigen escape. These toxin-based CAR T cells are distinct from conventional CAR design and could one day be used to deliver a poisonous blow to glioblastoma.

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Scientists develop new gene drive with a built-in genetic barrier

Scientists develop new gene drive with a built-in genetic barrier | Genetic Engineering - GEG Tech top picks | Scoop.it
CRISPR-based technologies offer enormous potential to benefit human health and safety, from disease eradication to fortified food supplies. As one example, CRISPR-based gene drives, which are engineered to spread specific traits through targeted populations, are being developed to stop the transmission of devastating diseases such as malaria and dengue fever.
BigField GEG Tech's insight:

CRISPR-based gene drives, which are designed to spread specific traits within targeted populations, are being developed to stop the transmission of devastating diseases such as malaria. However, many scientists and ethicists are concerned about the uncontrolled spread of these techniques. That's why scientists at the University of California, San Diego, and their colleagues have developed a genetic drive mechanism with a built-in genetic barrier designed to keep the mechanism in check. The researchers designed synthetic fly species that, when released in sufficient numbers, act as gene drives that can propagate locally and be reversed if necessary. The scientists describe their SPECIES (Synthetic Postzygotic barriers Exploiting CRISPR-based Incompatibilities for Engineering Species) development as a proof-of-concept innovation that could be translated to other species such as disease-vector insects. In theory, when SPECIES are deployed in the wild in sufficient numbers, they can spread in a controlled manner through a population and replace all of their wild counterparts as they spread. Using malaria as an example, SPECIES mosquitoes could be developed with a genetic component that makes them unable to transmit malaria.

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Sequential CD19-Directed CAR T-Cell Therapy and Allogeneic Transplant Provide Long-Term Responses in Pediatric B-ALL

Sequential CD19-Directed CAR T-Cell Therapy and Allogeneic Transplant Provide Long-Term Responses in Pediatric B-ALL | Genetic Engineering - GEG Tech top picks | Scoop.it
Sequential therapy with CD19.28ζ-directed CAR T cells followed by allogeneic hematopoietic stem cell transplant induced durable disease control in a significant population of children and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia.
BigField GEG Tech's insight:

CAR T cell therapies directed against CD19 have demonstrated high rates of pre-malignant responses in patients with relapsed B-cell acute lymphoblastic leukemia (B-ALL). However, long-term data on these therapies are limited. However, according to the results of a long-term follow-up analysis of a phase 1 study NCT01593696 published in the Journal of Clinical Oncology, sequential CAR-T cell therapy directed against CD19.28ζ followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) induces durable disease control in a significant population of pediatric and young adult patients with B-cell ALL. In the Phase 1 clinical trial, two doses of CAR T cell-based therapies were used, either an infusion of 1 x 10^6 CAR T cells/ kg administered on day 0, or an infusion of 3 x 10^6 CAR T cells/ kg administered on day 0. The maximum tolerated dose was defined as 1 x 10^6 CAR T cells / kg. Overall, 53 patients were enrolled in the study; 51 patients had B-ALL and 2 had diffuse large B-cell lymphoma. Complete responses were observed in 62% (n=31) of patients infused with CD19.28ζ directed CAR T cells (n=50).

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Replacing An Entire Faulty Gene To Reverse The Blood Disease β-Thalassemia

Replacing An Entire Faulty Gene To Reverse The Blood Disease β-Thalassemia | Genetic Engineering - GEG Tech top picks | Scoop.it
β-Thalassemia is caused in part by a mutated β-globin gene, so researchers developed a way to replace the entire mutated gene with a healthy version that restored and balanced protein production to a normal level.
BigField GEG Tech's insight:

β-thalassemia is a disease that is caused in part by a mutated β-globin HBB gene and involves a dangerous reduction in hemoglobin production, resulting in anemia, muscle weakness and fatigue. It affects one in 100,000 people worldwide, making it one of the most common genetic diseases in the world. Researchers at Stanford University have developed a gene therapy that replaces the entire mutated β-globin gene with a healthy version. They took stem cells from patients, cut out one of the two HBA genes that produces α-globin and replaced it with an HBB transgene using CRISPR-Cas9 technology, transfected the modified cells into the bodies of mice. The patients thus become their own donor. This technique allowed the α-globin promoter to be used to restore and balance β-globin production to a normal level. Now, the question is whether the corrective effects in single cells and mouse models translate into curative effects for human patients

 

https://www.nature.com/articles/s41591-021-01284-y

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Study offers localized treatment direction for a cause of Crohn's disease

Study offers localized treatment direction for a cause of Crohn's disease | Genetic Engineering - GEG Tech top picks | Scoop.it
People with Crohn's disease are typically treated with powerful anti-inflammatory medications that act throughout their body, not just in their digestive tract, creating the potential for unintended, and often serious, side effects.
BigField GEG Tech's insight:

Crohn's disease develops from a chronic inflammation of the digestive tract, often the small intestine. More than half a million people in the U.S. live with this disease, which may require repeated surgeries to remove irreversibly damaged bowel tissue. Current treatments for this disease are powerful anti-inflammatory drugs that work in all parts of the body and cause often severe side effects. However, Sundrud's team found that immune effector T cells in the small intestine have developed a molecular sensing mechanism to protect themselves from the toxic effects of high bile acid concentrations.

These T cells use an entire network of genes to safely interact with bile acids in the small intestine. The MDR1 gene, known as ABCB1, is activated when a large subset of CD4+ T cells enter the small intestine. Then, the MDR1 gene acts in transient T cells to suppress bile acid toxicity and inflammation in the small intestine. This pathway sometimes works less well in some people with Crohn's disease.

Based on this discovery, Sundrud's team developed a therapy based on CAR T cells that act in the small intestine and promote MDR1 expression and also play a role in activating a IL-10 gene. The result in mice was a localized detoxification of bile acids and a reduction in inflammation.

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Prime editing enables more precise correction of genetic problems than traditional CRISPR

Prime editing enables more precise correction of genetic problems than traditional CRISPR | Genetic Engineering - GEG Tech top picks | Scoop.it
The latest gene editing technology, prime editing, expands the "genetic toolbox" for more precisely creating disease models and correcting genetic problems, scientists say.
BigField GEG Tech's insight:

According to scientists at the Medical College of Georgia, a new edition of the CRISPR gene tool successfully suppressed the expression of Tspan2 gene involved in the differentiation of smooth muscle cells that help give strength and movement to organs and blood vessels. The researchers made a single-base change in the promoter region of Tspan2 and found that this inactivated the Tspan2 gene in the aorta and bladder without collateral damage unlike traditional CRISPR. This new editing only cuts a single strand of DNA unlike traditional editing which cuts double strands of DNA and makes unintended changes, which can be deadly to cells. Traditional CRISPR has 3 components: Cas9, molecular scissors that cuts both strands of DNA, guide RNA that directs the scissors to the right place and a repair model in case of problems. The new edition is composed of: Modified Cas9, named Cas9 nickase, which cuts a single strand of DNA, a complex called "main editor" with a reverse transcriptase and a PegRNA, the guide RNA of this new edition. One of the uses of this new CRISPR editing could be to create cell-specific knockout mice without extensive selection efforts that could also result in an exact model, for example, of the megacystis-microcolon-intestinal hypoperistalsis syndrome.

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New therapeutic vaccine uses patient's own tumor cells to aid in cancer destruction

New therapeutic vaccine uses patient's own tumor cells to aid in cancer destruction | Genetic Engineering - GEG Tech top picks | Scoop.it
Immunotherapy, which recruits the body's own immune system to attack cancer, has given many cancer patients a new avenue to treat the disease.
BigField GEG Tech's insight:

Treatments based on immunotherapy give hope to many people with cancer that they will finally be cured. However, some treatments can be very expensive, have side effects, or may only work on a small number of people. That's why researchers at the University of Chicago's Pritzer School of Molecular Engineering have developed a new therapeutic vaccine using a patient's own tumor cells that have been modified to secrete vascular endothelial growth factor and then irradiated so that the cells are dead before being reinjected. This vaccine would therefore train the patient's immune system to detect and eradicate cancer because, according to clinical trials, it stops the growth of melanoma tumors in mouse models. In addition, an immunological memory is set up thanks to the vaccine and leads to long-term effects because the vaccine would destroy the appearance of new tumor cells 10 months after the injection. The injection of the vaccine is done like a traditional vaccine. The advantages of this vaccine are that it would be more effective, less expensive and much safer.

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News: Promising Data From First-ever CRISPR Phage Therapy Trial

News: Promising Data From First-ever CRISPR Phage Therapy Trial | Genetic Engineering - GEG Tech top picks | Scoop.it
Locus Biosciences announced on Wednesday that it successfully completed the world's first clinical trial using a CRISPR-enhanced bacteriophage therapy. CRISPR-Cas3 enhanced the virus' natural ability to kill the E. coli bacteria behind urinary tract infections.
BigField GEG Tech's insight:

Locus Biosciences announced that it has completed the world's first clinical trial using a CRISPR-enhanced bacteriophage therapy in which CRISPR-Cas3 improved the natural ability of the virus to kill the E. coli bacteria behind urinary tract infections. The company decided to take a nuclear approach and to become the first company to combine both mechanisms, using both the lytic properties of bacteriophage and the DNA-destroying enzymatic properties of CRISPR-Cas3, thus increasing the killing capacity of naturally lytic phages. The co-founder and Scientific Director of Locus Biosciences explains that the study gives him hope that modified bacteriophages could one day become a new weapon in the fight against the growing threat of antimicrobial resistant strains of bacteria. During Phase I of the randomized, double-blind, placebo-controlled clinical trial called LBP-EC01, the research team did not see a single drug-related adverse event throughout the experiment. Phage therapy therefore has no impact at all on human cells. As a result, it is a much more accurate tool for killing bacteria than broad-spectrum antibiotics or other therapies currently in use. More importantly, data suggest that it is safe for humans, even at high doses. Phase II will therefore begin shortly.

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Researchers identify protein that may represent a safer treatment target for pancreatic cancer

Researchers identify protein that may represent a safer treatment target for pancreatic cancer | Genetic Engineering - GEG Tech top picks | Scoop.it
Researchers from Queen Mary University of London, have identified a protein that may represent a novel therapeutic target for the treatment of pancreatic cancer.
BigField GEG Tech's insight:

Pancreatic ductal adenocarcinoma (PDAC) is the most common type of pancreatic cancer and has the lowest survival rate of all common cancers. Only about 7% of people diagnosed with this type of cancer in the UK survive their cancer for 5 years or more. However, a new protein called CEACAM7 has been identified by researchers at Queen Mary University. It could be a new therapeutic target for the treatment of PDAC which is the most common type of pancreatic cancer. In this study, the researchers developed a novel CAR T cell therapy using part of an anti-CEACAM7 antibody from Professor Brad Nelson (British Columbia, Canada). They then modified the killer T cells and presented on their surface this new CAR protein that recognizes and binds to CEACAM7, directing the killer T cells to kill only the cells with CEACAM7. Using this protein as a target, the researchers were able to create a CAR T cell therapy that killed pancreatic cancer cells in a pre-clinical model.  

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New Screening Approach Reveals Novel Regulators of Microcephaly

New Screening Approach Reveals Novel Regulators of Microcephaly | Genetic Engineering - GEG Tech top picks | Scoop.it
Researchers combine organoids, CRISPR-Cas9, and cellular barcoding technologies to identify genes that influence brain size.
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Organoids can be used to study human diseases, but they are often difficult to use, especially when evaluating several candidate genes that underlie a particular condition. A team at the Institute for Molecular Biotechnology in Vienna has therefore found a way around this problem. Their approach combines brain organoids with two other technologies that are CRISPR-Cas9 to knock out specific genes and DNA barcodes to track individual cells and their progeny. This approach has been named CRISPR-LIneage tracing at Cellular Resolution in Heterogeneous Tissue and found 12 genes with a specific function in the tissue once the organoid began to take the form of a small brain. Among these 12 genes, the researchers showed that the IER3IP1 gene mutation results in abnormally small organoids because their neural progenitor cells differentiate into neurons before the organoids develop properly. The IER3IP1 peptide appears to block premature differentiation by promoting the secretion of extracellular matrix proteins that support tissue integrity and proliferation of neural progenitors.  

 

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New cell therapy can boost immunotherapy against breast cancer

New cell therapy can boost immunotherapy against breast cancer | Genetic Engineering - GEG Tech top picks | Scoop.it

Boosting immune system T cells to effectively attack solid tumors, such as breast cancers, can be done by adding a small molecule to a treatment procedure called chimeric antigen receptor-T (CAR-T) cell therapy, according to a study by researchers at the UNC Lineberger Comprehensive Cancer Center.

BigField GEG Tech's insight:

CAR T cell immunotherapies are more effective as a treatment for patients with leukemia or B cell lymphomas because once they are re-injected into the patient, they migrate and lodge in the bone marrow and other organs that make up the lymphatic system. However, for solid tumours, such as breast cancer, the CAR T cells have difficulty migrating to the tumour because of the microenvironment that surrounds it. Recently, a study conducted by researchers at the UNC Lineberger Comprehensive Cancer Center, shows that adding a small molecule to the CAR T cell-based treatment could stimulate the Th17 and Tc17 cells of the immune system to effectively attack solid tumours. To stimulate the accumulation of these Th17 and Tc17 cells in the vicinity of solid tumours, the research team discovered that the stimulator of interferon agonist (STING) genes, cGAMP, activates the human STING and is known to stimulate the human immune system. The various experiments showed that mice injected with cGAMP showed increased proliferation of T cells and these cells migrated to the tumour site. The end result was a significant decrease in tumour growth and improved survival.  

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Building better CAR-T therapies

Building better CAR-T therapies | Genetic Engineering - GEG Tech top picks | Scoop.it
The current technique requires a person’s own cells, but using the cells of healthy donors could allow more people to benefit.
BigField GEG Tech's insight:

Currently, most people with cancer could be treated with CAR T cell therapy but they don't benefit from it. The main reason is that this treatment is difficult to produce. The average delay between donation and receipt of therapy is more than three weeks. For people with rapidly proliferating diseases, such as acute leukemia, this may be too long to wait. The current technique requires a person's own cells, but the use of cells from healthy donors could allow more people to benefit. However, donor T cells can identify the body of the person receiving the treatment as foreign and attack it, triggering Graft-Versus-Host Disease, which can be fatal, or the foreign T cells can be eliminated by the person's immune system before they can attack the cancer. To counter these problems, biotech company Allogene Therapeutics of southern San Francisco, California, has genetically engineered T-cells to remove a protein known as CD52 from their surfaces. Antibodies that help destroy the cells that carry the surface protein are then administered to the person, depleting his or her own white blood cells that could otherwise kill the modified CAR T cells. And to protect against GVHD, the T-cell receptor on the modified cells can be altered, preventing them from attacking the person's own cells.

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T cells engineered to target senescence

T cells engineered to target senescence | Genetic Engineering - GEG Tech top picks | Scoop.it
CAR T cells that target senescent cells combat disease in mouse models.
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Senescence is a hallmark of cellular ageing and contributes to many diseases. A new method enabling immune cells to target senescent cells might offer improved therapeutic options.

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