Vectorology - GEG Tech top picks
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MGMT enrichment and second gene co-expression in hematopoietic progenitor cells using separate or dual-gene lentiviral vectors

MGMT enrichment and second gene co-expression in hematopoietic progenitor cells using separate or dual-gene lentiviral vectors | Vectorology - GEG Tech top picks | Scoop.it
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Scientists compared selection and expression efficacies in cells cotransduced with separate single-gene MGMT and GFP lentivectors to those obtained with dual-gene vectors employing either internal ribosome entry site (IRES) or 2A elements for co-expression strategies. The highest dual-gene expression (MGMT+GFP+) percentages were obtained with the 2A dual-gene vector, but half of the resulting gene products existed as fusion proteins. Equivalent MGMT expression levels were obtained with each strategy, but GFP expression levels derived from the IRES dual-gene vector were significantly lower.


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Viral Vectors and Plasmid DNA Manufacturing Market Report, 2018-2030

Viral Vectors and Plasmid DNA Manufacturing Market Report, 2018-2030 | Vectorology - GEG Tech top picks | Scoop.it
Viral Vectors and Plasmid DNA Manufacturing Market, 2030
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The Viral Vectors and Plasmid DNA Manufacturing Market (2nd Edition), 2018-2030 report offers a comprehensive study of the current scenario of manufacturing of viral and non-viral vectors that are primarily used for the development of gene therapies and T-cell therapies. The study features an in-depth analysis, highlighting the capabilities of a diverse set of players, covering both contract manufacturers and companies with in-house capabilities.

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CRISPR/Cas9-Mediated Genome Editing for Huntington’s Disease

CRISPR/Cas9-Mediated Genome Editing for Huntington’s Disease | Vectorology - GEG Tech top picks | Scoop.it
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This chapter describes the potential use of viral-mediated gene transfer in the central nervous system for genome editing in the context of Huntington’s disease. Here, the scientist provide protocols that cover the design of various genome editing strategies, the cloning of CRISPR/Cas9 elements into lentiviral vectors, and the assessment of cleavage efficiency, as well as potential unwanted effects.

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Gene therapy could free some people from a lifetime of blood transfusions

Gene therapy could free some people from a lifetime of blood transfusions | Vectorology - GEG Tech top picks | Scoop.it
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
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Correcting a genetic mutation lets beta thalassemia patients make healthy blood cells.

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Gene therapy researchers find viral barcode to cross the blood-brain barrier

Gene therapy researchers find viral barcode to cross the blood-brain barrier | Vectorology - GEG Tech top picks | Scoop.it
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UNC School of Medicine scientists led by Aravind Asokan, PhD, reveal how certain gene-carrying AAV vectors can penetrate the brain more efficiently to treat brain and spinal cord conditions, while reducing liver payload.
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System for the live tracking of lentiviral vectors - VIDEO

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The ANCHOR breakthrough technology developed by NeoVirTech allows real time visualisation and quantification of virus and viral vector infection/transduction and replication in living cells. This innovative system allows to tag the virus genomes and to visualize their cycle with an extreme accuracy.
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ITAV | Première visualisation en direct d’un lentivirus au sein de cellules humaines vivantes

ITAV | Première visualisation en direct d’un lentivirus au sein de cellules humaines vivantes | Vectorology - GEG Tech top picks | Scoop.it
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NeoVirTech et GEG Tech annoncent la validation de la technologie ANCHORTM pour une forme modifiée du virus du SIDA. La technologie ANCHORTM développée par NeoVirTech permet de marquer l’ADN de virus et de visualiser son évolution en temps réel au sein de cellules vivantes. Il a été intégré avec succès par GEG Tech dans un virus dérivé du SIDA.
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Sleeping Beauty Insertional Mutagenesis in Mice Identifies Drivers of Steatosis-Associated Hepatic Tumors

Sleeping Beauty Insertional Mutagenesis in Mice Identifies Drivers of Steatosis-Associated Hepatic Tumors | Vectorology - GEG Tech top picks | Scoop.it
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In this study, the scientists performed a forward genetic screen using Sleeping Beauty (SB) transposon insertional mutagenesis in mice treated to induce hepatic steatosis and compared the results to human HCC data. In humans, they determined that steatosis increased the proportion of female HCC patients, a pattern also reflected in mice. Their genetic screen identified 203 candidate steatosis-associated HCC genes, many of which are altered in human HCC and are members of established HCC-driving signaling pathways.
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In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation

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In vivo delivery of a Cas9-based epigenetic gene activation system ameliorates disease phenotypes in mouse models of type I diabetes, acute kidney injury, and muscular dystrophy
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Dynamics of Indel Profiles Induced by Various CRISPR/Cas9 Delivery Methods 

Dynamics of Indel Profiles Induced by Various CRISPR/Cas9 Delivery Methods  | Vectorology - GEG Tech top picks | Scoop.it
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In this study the scientists review the most commonly used indel detection methods and using a robust, sensitive, and cost efficient Indel Detection by Amplicon Analysis method, they have investigated the impact of the most commonly used CRISPR/Cas9 delivery formats, including lentivirus transduction, plasmid lipofection, and ribo nuclear protein electroporation, on the dynamics of indel profile formation. They observe rapid indel formation using RNP electroporation, especially with synthetic stabilized gRNA, as well as long-term decline in overall indel frequency with lipofectamine-based, plasmid transfection methods. Most methods reach peak editing on day 2–3 postdelivery. Furthermore, they find relative increase in frequency of larger size indels (>6 bp) under condition of persistent editing using stably integrated lentiviral gRNA and Cas9 vectors.
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French Biotech Raises €19M for a Gene Therapy Trial in Retinitis Pigmentosa

French Biotech Raises €19M for a Gene Therapy Trial in Retinitis Pigmentosa | Vectorology - GEG Tech top picks | Scoop.it
The European Biotech News Website
BigField GEG Tech's insight:
Horama has raised a second fundraising round that will support its lead candidate, a gene therapy for retinitis pigmentosa, through clinical trials.
HORA-PDE6B is intended to cure retinitis pigmentosa in patients that develop the disease because of a mutation in the PDE6β gene, which amounts to around 5,000 people in the US and Europe. The gene therapy consists of a recombinant adeno-associated virus (AAV) in which the viral DNA is replaced by a functional copy of the PDE6β gene.
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Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1 | Vectorology - GEG Tech top picks | Scoop.it
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Although gene therapy has been proposed for a variety of genetic disorders, including severe combined immunodeficiency, it has not yet found routine use in the clinic, in part because of potential complications. To help pave the way for safer translation of such gene therapy, Schiroli et al. studied potential approaches to it in mouse models of severe combined immunodeficiency. The authors systematically analyzed the outcomes of using different approaches to conditioning, different numbers of gene-edited cells, different techniques for editing the faulty gene, and other aspects of the technology to find the safest and most effective method.
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Absence of Replication-Competent Lentivirus in the Clinic: Analysis of Infused T Cell Products - Cell

Absence of Replication-Competent Lentivirus in the Clinic: Analysis of Infused T Cell Products - Cell | Vectorology - GEG Tech top picks | Scoop.it
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The safety of lentiviral vectors is a factor in their acceptance as clinical therapies. In this issue of Molecular Therapy, Cornetta et al. (2017) screened 460 cell products for replication-competent lentivirus (RCL); none were positive. The low risk of RCL suggests that revisions to U.S. FDA testing guidelines are warranted.

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Oxford BioMedica could pocket $100m on Novartis CAR-T cell success

Oxford BioMedica could pocket $100m on Novartis CAR-T cell success | Vectorology - GEG Tech top picks | Scoop.it
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Oxford BioMedica saw its share price jump 21% after the US FDA approved Kymriah, the gene modified cell therapy made with the firm’s lentiviral vectors.
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A Versatile Safeguard for Chimeric Antigen Receptor T-Cell Immunotherapies

A Versatile Safeguard for Chimeric Antigen Receptor T-Cell Immunotherapies | Vectorology - GEG Tech top picks | Scoop.it
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Here, the scientists report the development of an alternative safeguard for chimeric antigen receptor T-cell therapies. They have incorporated a safeguard component into a conventional CAR architecture that enables tight control over CAR T-cell activity and has the potential to improve their safety profile in clinical settings. In addition, the CubiCAR architecture is compatible with multiple scFvs designed against different targets, thus bearing the potential to improve the safety of CAR T-cell immunotherapies for a broad range of patients.

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Toulouse : première visualisation dynamique du virus du Sida dans des cellules humaines - France 3 

Toulouse : première visualisation dynamique du virus du Sida dans des cellules humaines - France 3  | Vectorology - GEG Tech top picks | Scoop.it
BigField GEG Tech's insight:

NeoVirTech and GEG Tech are proud to announce the first real-time visualization of HIV-1 vector in living cells. The ANCHORTM technology developed by NeoVirTech enables autonomous fluorescent tagging of any DNA fragment to follow its behavior in living cells. It was successfully integrated by GEG Tech in their Lenti-ONE vector based on HIV-1 virus. This result will i) open new research paths in the fight against HIV/AIDS, and ii) accelerate innovation in genetic engineering fields exploiting  HIV-1 derived vectorization systems.

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Oxford BioMedica, Bioverativ to Develop Lentiviral Vectors for Hemophilia Gene Therapy

Oxford BioMedica, Bioverativ to Develop Lentiviral Vectors for Hemophilia Gene Therapy | Vectorology - GEG Tech top picks | Scoop.it
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Bioverativ has agreed to license Oxford BioMedica’s LentiVector® Enabled technology, as well as its industrial-scale manufacturing technology for up to $105 million-plus
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Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing | Vectorology - GEG Tech top picks | Scoop.it
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Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.
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JCI Insight - Enhancing CAR T cell persistence through ICOS and 4-1BB costimulation

JCI Insight - Enhancing CAR T cell persistence through ICOS and 4-1BB costimulation | Vectorology - GEG Tech top picks | Scoop.it
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In this study, scientists demonstrated that ICOS and 4-1BB ICD increase CARs efficacy in solid tumor models over current 4-1BB–based CAR and are promising therapeutics for clinical testing.
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First-ever live tracking of HIV-1 based lentiviral vectors in human cells

First-ever live tracking of HIV-1 based lentiviral vectors in human cells | Vectorology - GEG Tech top picks | Scoop.it
BigField GEG Tech's insight:
Technology breakthrough: visualization of lentivirus genomes in living cells using ANCHOR technology.

We are proud to announce that we obtain great results through a collaborative project with NeoVirTech, a company developing auto fluorescent virus for imaging and screening applications. GEG Tech has validated the auto-fluorescent ANCHOR tagging of lentiviral vectors (HIV-1 derived) for imaging and screening applications. Both integrating and non-integrating mutants can be visualized thanks to the ANCHOR technology, allowing the first-ever live tracking of lentiviral genomes, integrated or not, the observation of vector genome dynamics (see video) and the exact determination of copy number by cell (see illustration and legend). 

 This new system allows the designing of innovative lentiviral vectors, and opens new ways for gene transfer tool users as well as for R&D in vectorology and virology. 

GEG Tech will make available this technology to its clients and will offer new services in order to exploit this powerful solution. GEG tech remains committed to its customers to design new gene transfer solutions and deliver breakthrough innovations. 

Further details in the coming weeks...

To know more about GEG Tech products: https://www.geg-tech.com. About NeoVirTech: http://neovirtech.com 

Legend Images show transduced cells with ANCHOR HIV-1 derived lentivirus 24h PT (left) and 48hPT (right). Fluorescent spots correspond to the position of lentivirus genomes. 
For example, 44 vector genomes copies can be detected in the cell nucleus 24hPT.
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bluebird bio Acquires Durham, NC, Manufacturing Site for Lentiviral Vector Production 

bluebird bio Acquires Durham, NC, Manufacturing Site for Lentiviral Vector Production  | Vectorology - GEG Tech top picks | Scoop.it
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Bluebird bio said today it has acquired a manufacturing facility in Durham, NC, from an undisclosed seller for $11.5 million, with the goal of producing lentiviral vectors for the company’s cell and gene therapies.
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CD22-targeted CAR T cells induce remission in B-ALL that is naive or resistant to CD19-targeted CAR immunotherapy

CD22-targeted CAR T cells induce remission in B-ALL that is naive or resistant to CD19-targeted CAR immunotherapy | Vectorology - GEG Tech top picks | Scoop.it
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Researchers report promising results in a Phase 1 trial testing a new cell therapy using chimeric antigen receptor (CAR) T-cell technology on patients suffering from a treatment-resistant form of leukemia. The study, which successfully treated even cancers that had resisted a previous CAR T immunotherapy, was published in Nature Medicine
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Regeneration of the entire human epidermis using transgenic stem cells - Nature

Regeneration of the entire human epidermis using transgenic stem cells - Nature | Vectorology - GEG Tech top picks | Scoop.it
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Junctional epidermolysis bullosa (JEB) is a severe and often lethal genetic disease caused by mutations in genes encoding the basement membrane component laminin-332. Surviving patients with JEB develop chronic wounds to the skin and mucosa, which impair their quality of life and lead to skin cancer. Here the authors show that autologous transgenic keratinocyte cultures regenerated an entire, fully functional epidermis on a seven-year-old child suffering from a devastating, life-threatening form of JEB. The proviral integration pattern was maintained in vivo and epidermal renewal did not cause any clonal selection. Clonal tracing showed that the human epidermis is sustained not by equipotent progenitors, but by a limited number of long-lived stem cells, detected as holoclones, that can extensively self-renew in vitro and in vivo and produce progenitors that replenish terminally differentiated keratinocytes. This study provides a blueprint that can be applied to other stem cell-mediated combined ex vivo cell and gene therapies.
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A Novel construction of lentiviral vectors for eliminating tumorigenic cells from pluripotent stem cells

A Novel construction of lentiviral vectors for eliminating tumorigenic cells from pluripotent stem cells | Vectorology - GEG Tech top picks | Scoop.it
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The risk of tumor formation poses a challenge for human pluripotent stem cell (hPSC)‐based transplantation therapy. Specific and total elimination of tumorigenic hPSCs by suicide genes has not bee
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Turning Brain Cells into Skin Cells

Turning Brain Cells into Skin Cells | Vectorology - GEG Tech top picks | Scoop.it
OCT4 impedes cell fate redirection by the melanocyte lineage master regulator MITF in mouse ESCs
BigField GEG Tech's insight:
A new study published in Nature Communications reveals that it is possible to repurpose the function of different mature cells across the body — and harvest new tissue and organs from these cells.
The research tracks the transformation of genetically manipulated cells into melanocytes, which are responsible for the production of skin pigment and essential to the body's auditory system.
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Comparison of ZFNs versus CRISPR specific nucleases for genome edition of the Wiskott-Aldrich Syndrome locus

Comparison of ZFNs versus CRISPR specific nucleases for genome edition of the Wiskott-Aldrich Syndrome locus | Vectorology - GEG Tech top picks | Scoop.it
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In this work, the scientists have designed different WAS gene-specific CRISPR/Cas9 systems and compared their efficiency and specificity using K562 cells as a cellular model and plasmid nucleofection or integrative-deficient Lentiviral Vectors (IDLVs) for delivery. The different CRISPR/Cas9 and ZFNs SNs showed similar efficiency using plasmid nucleofection for delivery. However, dual IDLVs expressing ZFNs were more efficient than dual IDLVs expressing Cas9 and gRNA or that all-in-one IDLVs, expressing Cas9 and gRNA in the same vector. They also showed a similar behavior of heterodimeric ZFNs and CRISPR/Cas9 for Homology-Directed (HD) gene knock-in strategies, with 88% and 83% of the donors inserted in the WAS locus respectively while using homodimeric ZFNs only 45% of the insertions were on target. In summary, their data indicates that CRISPR/Cas9 and heterodimeric ZFNs are both good alternatives to further develop SNs-based gene therapy strategies for WAS. However IDLVs delivery of WAS-specific heterodimeric ZFNs was the best option of all systems compared in this study.

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