Previous efforts to circumvent the problem of the size limitation imposed by the AAV vector have focused mainly on altering the candidate genes into ‘mini-expression cassettes’ suitable for ...
First demonstration of single AAV muscle editing in non-human primates: NanoCas achieved in vivo editing efficiencies of up to 30% when targeting dystrophin in the skeletal muscle of cynomolgus ...
Henrickson A, Ding X, Seal A, Qu Z, Tomlinson L, Forsey J, Gradinaru V, Oka K, Bemeler B. Characterization and quantification of adeno-associated virus capsid-loading states by multi-wavelength ...
That therapy uses an adeno-associated virus (AAV) as a vector rather than a lentivirus, a vector type that features in approved gene therapies such as Roche's Luxturna and Novartis' Zolgensma.
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 ...
Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...
Genethon & Eukarÿs enter strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies: Evry, France Friday, January 24, 2025, ...
Merck's expertise spans viral vector types including adeno-associated virus, lentivirus and adenovirus. The company provides contract testing services and extensive contract development and ...