Current lentiviral vector systems (the third generation ... systemic delivery of these vectors remains a major hurdle that has stymied gene therapy applications. But there’s movement.
Co-expressing NPY and its inhibitory receptor Y2 in excitatory hippocampal cells allows inhibitory autoregulation of ...
Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, skin, and skeleton with no ...
was seen in a patient treated with Skysona that probably arose as a result of the lentiviral vector used to deliver the gene therapy. There's a well-recognised risk that insertion of a gene ...
From Orchard’s Lenmeldy to Vertex and CRISPR Therapeutics’s Casgevy, Pharmaceutical Technology lists the top ten priciest ...
Researchers from Children's Hospital of Philadelphia (CHOP) have tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that ...
The lentiviral vector delivers the SGSH gene to bone marrow cells ... Professor Bobby Gaspar, Chief Scientific Officer of Orchard Therapeutics said: “Stem cell gene therapy has shown promising effects ...
Analyst Cory Jubinville, PhD of LifeSci Capital maintained a Buy rating on Rocket Pharmaceuticals (RCKT – Research Report), retaining the ...
One of the original pioneers in cell and gene therapy, OXB has more than 25 years ... and biotechnology companies, providing viral vector development and manufacturing expertise in lentivirus, ...
Its multi-platform development approach applies the lentiviral vector and adeno-associated viral vector gene therapy platforms. Its clinical program is an LVV-based gene therapy for the treatment ...
More recently, the Vector Development Laboratory merged with our group, and we have expanded our services to offer AAV, first-generation adenovirus (FGAd), helper-dependent adenovirus (HDAd), ...