Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

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 …

Replacing or editing disease-causing mutations holds great promise for treating many
human diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has …

The sponsor of this clinical study, Audentes Therapeutics (which was acquired by Japan's
Astellas Pharma in 2019), had generated impressive efficacy studies in a canine model of …

Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …

Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we …

Background X-linked myotubular myopathy is a rare, life-threatening, congenital muscle
disease observed mostly in males, which is caused by mutations in MTM1. No therapies are …

Immune responses in gene therapy with adeno-associated virus (AAV) vectors have been
the object of almost two decades of study. Although preclinical models helped to define and …

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 …

By virtue of their transformational power, the technologies at the core of the gene therapy
community have brought widespread public attention to the unique burden of previously …