Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

(MENAFN- EIN Presswire) Leading Pharmaceutical companies are advancing the AAV vector gene therapy pipeline and its future growth potential. LAS VEGAS , NV, UNITED STATES, February 7, 2025 ...

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

The candidate in question is VY9323, an AAV gene therapy combining a highly potent pri-miRNA against superoxide dismutase 1 ...

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

Astellas has invested heavily in novel technologies that are not necessarily the most successful or popular in the industry.

The Tokyo-based biotech firm expects RV-001 to be able to alter light sensitivity for patients with limited or no vision regardless of genotype.

In addition, first-generation CRISPR systems (Cas9 and Cas12a) are too large for efficient in vivo delivery via a single adeno-associated viral (AAV) vector ... the PCSK9 gene in mouse liver ...

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

The nano-complexes released the loaded-AAV intracellularly ... approach to viral vector treatments, which are currently limited by neutralizing antibodies. Gene therapy is a treatment method ...