Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

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 …

Genome‐editing therapeutics are poised to treat human diseases. As we enter clinical trials
with the most promising CRISPR‐Cas9 and CRISPR‐Cas12a (Cpf1) modalities, the risks …

Glycogen storage disease type II or Pompe disease is a severe neuromuscular disorder
caused by mutations in the lysosomal enzyme, acid α-glucosidase (GAA), which result in …

Early preclinical studies in rodents and other species did not reveal that vector or transgene
immunity would present a significant hurdle for sustained gene expression. While there was …

Primary mitochondrial diseases represent some of the most common and severe inherited
metabolic disorders, affecting~ 1 in 4,300 live births. The clinical and molecular diversity …

Myotonic dystrophy type I (DM1) is a multisystemic autosomal-dominant inherited human
disorder that is caused by CTG microsatellite repeat expansions (MREs) in the 3 …

Pompe disease (glycogen storage disease type II) is caused by mutations in acid α-
glucosidase (GAA) resulting in lysosomal pathology and impairment of the muscular and …

Glycogen storage disorders (GSDs) are inherited disorders of metabolism resulting from the
deficiency of individual enzymes involved in the synthesis, transport, and degradation of …