Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo
gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer …

This study examines transgene expression and biodistribution of adeno-associated virus
(AAV) pseudotyped 1–9 after tail vein (TV) injection in male mice. Using a cytomegalovirus …

Recombinant adeno-associated viral (AAV) vectors have rapidly advanced to the forefront of
gene therapy in the past decade. The exponential progress of AAV-based vectors has been …

Adeno-associated virus (AAV) serotypes differ broadly in transduction efficacies and tissue
tropisms and thus hold enormous potential as vectors for human gene therapy. In reality …

Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Site-specific gene addition can allow stable transgene expression for gene therapy. When
possible, this is preferred over the use of promiscuously integrating vectors, which are …

Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …

Chronic hepatitis B (CHB) is a global health challenge that can result in significant liver-
related morbidity and mortality. Despite a prophylactic vaccine being available, patients …

Adeno-associated virus (AAV) vectors efficiently transduce various cell types and can
produce long-term expression of transgenes in vivo. Although AAV vector genomes can …