Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...

Analyses and Examinations Integration-site analysis was performed, according to published methods, 14,15 on the peripheral blood of the patients who were treated with eli-cel to determine patterns of ...