Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Abstract Background Patients with the Crigler–Najjar syndrome lack the enzyme uridine
diphosphoglucuronate glucuronosyltransferase 1A1 (UGT1A1), the absence of which leads …

Neutralizing antibodies to adeno-associated virus (AAV) vectors are highly prevalent in
humans,, and block liver transduction,–and vector readministration; thus, they represent a …

Gene therapy mediated by recombinant adeno-associated virus (AAV) vectors is a
promising treatment for systemic monogenic diseases. However, vector immunogenicity …

Pompe disease is a rare and deadly muscle disorder. As a clinical entity, the disease has
been known for over 75 years. While an optimist might be excited about the advances made …

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

Glycogen synthase 1 (GYS1), the rate-limiting enzyme in muscle glycogen synthesis, plays a
central role in energy homeostasis and has been proposed as a therapeutic target in …

Cachexia is a wasting disorder that accompanies many chronic diseases including cancer
and results from an imbalance of energy requirements and energy uptake. In cancer …

Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …