Vectorology - GEG Tech top picks
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Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration | Vectorology - GEG Tech top picks | Scoop.it
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Immunogenicity of AAV vectors renders repeated AAV dosing ineffective. Here the authors show that coadministration of nanoparticle-encapsulated rapamycin overcomes AAV immunogenicity through Treg induction, enabling efficient AAV redosing in mice and nonhuman primates.

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The IS2 Element Improves Transcription Efficiency of Integration-Deficient Lentiviral Vector Episomes

The IS2 Element Improves Transcription Efficiency of Integration-Deficient Lentiviral Vector Episomes | Vectorology - GEG Tech top picks | Scoop.it
BigField GEG Tech's insight:

Integration-defective lentiviral vectors (IDLVs) have become an important alternative
tool for gene therapy applications and basic research. Unfortunately, IDLVs show lower
transgene expression as compared to their integrating counterparts. In this study,
we aimed to improve the expression levels of IDLVs by inserting the IS2 element, which
harbors SARs and HS4 sequences, into their LTRs (SE-IS2-IDLVs). Contrary to our expectations,
the presence of the IS2 element did not abrogate epigenetic silencing by histone deacetylases.

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At the Bench-Spinal Cord Injury: Experimental Gene Therapy Restores Movement After Spinal Cord Injury in Rats

At the Bench-Spinal Cord Injury: Experimental Gene Therapy Restores Movement After Spinal Cord Injury in Rats | Vectorology - GEG Tech top picks | Scoop.it
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In an animal model of spinal cord injury, 2.5 weeks of exposure to a novel gene therapy led to improvements in motor tasks; longer exposure to the gene therapy (eight weeks) led to more significant improvement — the rats could perform more complex functions such as grasping and retrieving sugar pellets with their paws.

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Cambridge Biotech to Launch CAR-T Manufacturing Technique

Cambridge Biotech to Launch CAR-T Manufacturing Technique | Vectorology - GEG Tech top picks | Scoop.it

Torque Therapeutics, a biotech company in Cambridge, Massachusetts, announced its high-efficiency T-cell manufacturing process called Slipstream.

BigField GEG Tech's insight:

Proud to see one of our users at the cusp of implementing its ambitious CAR-T manufacturing project!

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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
BigField GEG Tech's insight:

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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BigField GEG Tech's insight:
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
BigField GEG Tech's insight:
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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AVROBIO Reports Encouraging Preliminary Data for Phase 1/2 Trial Testing Gene Therapy in Fabry Patients

AVROBIO Reports Encouraging Preliminary Data for Phase 1/2 Trial Testing Gene Therapy in Fabry Patients | Vectorology - GEG Tech top picks | Scoop.it
AVROBIO announced positive preliminary data for its Phase 1/2 trial testing AVR-RD-01 gene therapy in patients with Fabry disease.
BigField GEG Tech's insight:

AVROBIO, a biotechnology company developing single-dose gene therapies for rare diseases, announced positive preliminary data for its Phase 1/2 clinical trial testing AVR-RD-01 gene therapy in patients with Fabry disease.

AVR-RD-01 consists of a lentiviral vector (transporter) containing a functional copy of the GLA gene, the gene mutated in Fabry disease, that is used to modify hematopoietic stem cells of the patients. 

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Amygdala GluN2B-NMDAR dysfunction is critical in abnormal aggression of neurodevelopmental origin induced by St8sia2 deficiency

Amygdala GluN2B-NMDAR dysfunction is critical in abnormal aggression of neurodevelopmental origin induced by St8sia2 deficiency | Vectorology - GEG Tech top picks | Scoop.it
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Beautiful example using a lentiviral vector designed to investigate the morphology of lateral amygdala pyramidal neurons. 

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NIH-funded researchers reverse congenital blindness in mice | National Eye Institute

NIH-funded researchers reverse congenital blindness in mice | National Eye Institute | Vectorology - GEG Tech top picks | Scoop.it
Restoration of vision after de novo genesis of rod photoreceptors in mammalian retinas - Nature

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BigField GEG Tech's insight:

Researchers funded by the National Eye Institute (NEI) have reversed congenital blindness in mice by changing supportive cells in the retina called Müller glia into rod photoreceptors. The findings advance efforts toward regenerative therapies for blinding diseases such as age-related macular degeneration and retinitis pigmentosa. A report of the findings appears online today in Nature. NEI is part of the National Institutes of Health.

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Electric Shock Allows for CRISPR Gene Editing Without a Viral Vector

Electric Shock Allows for CRISPR Gene Editing Without a Viral Vector | Vectorology - GEG Tech top picks | Scoop.it
Reprogramming human T cell function and specificity with non-viral genome targeting - Nature
BigField GEG Tech's insight:

quick zap of electricity makes T cells more receptive to taking in new genetic material and gene-editing reagents, researchers report July 11 in Nature. The discovery could expedite protocols for creating immunotherapies to treat a range of cancers.

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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
BigField GEG Tech's insight:

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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BigField GEG Tech's insight:
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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