This intrinsic property of AAV to undergo intermolecular ... from a minimal simian virus 40 (SV40) promoter and an increase in expression of 200-fold from the ITR in a vector without a promoter.

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

After endosomal escape, AAVs can also successfully transduce the cell and deliver their viral genome to the nucleus, where the transgene is expressed. Any misfolded protein encoded by the transgene ...

Oxford, UK - 5 February 2025: OXB (LSE: OXB), a quality and innovation-led cell and gene therapy CDMO, today announces the ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

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.

Viralgen and the CTNNB1 Foundation have come together to manufacture an investigational gene therapy designed to treat CTNNB1 ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Thermo Fisher’s office in Cambridge, Massachusetts. Image credit: Shutterstock/Tada Images. Thermo Fisher is cutting the headcount at two of its US facilities by 300 as the company continues to ...

CRISPR-based gene editing seems to be feasible for the development of new therapeutic strategies for OA treatment. Adeno-associated virus, which expressed CRISPR/Cas9 components to target each of the ...