The history of brain drug delivery is reviewed beginning with the first demonstration, in 1914,
that a drug for syphilis, salvarsan, did not enter the brain, due to the presence of a blood …

On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …

The development of liver-based adeno-associated virus (AAV) gene therapies is facing
concerns about limited efficiency and durability of transgene expression. We evaluated …

Hutchinson–Gilford progeria syndrome (HGPS or progeria) is typically caused by a
dominant-negative C• G-to-T• A mutation (c. 1824 C> T; p. G608G) in LMNA, the gene that …

Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy
and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine …

While there has been substantial progress and notable achievements in the use of adeno-
associated virus (AAV) gene therapy vectors for treatment of rare diseases, setbacks related …

Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential
for treating and curing genetic disorders. For otherwise incurable indications, including most …

Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for
both monogenic and complex acquired disorders. The success of AAV gene transfer …

Two decades after the initial gene therapy trials and more than 1700 approved clinical trials
worldwide we not only have gained much new information and knowledge regarding gene …

Viral vectors are promising tools for gene therapy and vaccines. Viral vector-based vaccines
can enhance immunogenicity without an adjuvant and induce a robust cytotoxic T …