FEATURE STORY ● Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy GENE THERAPY ● Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model ● Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 RARE DISEASES ● Characterization and treatment of congenital thrombotic thrombocytopenic purpura ● Targeting angiogenesis in…..
The genome editing system CRISPR has become a hugely important tool in medical research. The targeting system can travel to different points on the genome, guided by a short sequence of RNA, where the DNA-cutting enzyme Cas9 then makes the desired edits. However, CRISPR-Cas9 remains limited in the number of…..
Gene Therapy is a rapidly and obviously evolving field that is now definitively recognized as a well-acknowledged major medical and scientific area. BioPharmAnalyses is proud to announce the launch of its new global commercial report "Landscape in Gene Therapy Companies". This is the first such report that provides you with an actually updated information and an accurate international mapping…..
Vingt-cinq ans après les premiers essais de thérapie génique chez l’homme, Serge Braun, directeur scientifique de l’AFM-Telethon (photo), fait le point sur la situation actuelle de ces nouveaux traitements qui intéressent maintenant un nombre croissant de big pharma et de biotech. BioPharmAnalyses : Quelles sont les technologies les plus utilisées…..
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