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 …

With the recent development of CRISPR technology, it is becoming increasingly easy to
engineer the genome. Genome-editing systems based on CRISPR, as well as transcription …

Over the past decade, vectors derived from adeno-associated virus (AAV) have established
themselves as a powerful tool for in vivo gene transfer, allowing long-lasting and safe …

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 …

Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the
lives of patients with certain genetic disorders by increasing or restoring function to affected …

Recently, adeno-associated virus (AAV)-mediated gene therapies have attracted clinical
interest for treating neurodegenerative diseases including spinal muscular atrophy (SMA) …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

Pompe disease, also known as glycogen storage disease type II, is caused by the lack or
deficiency of a single enzyme, lysosomal acid alpha-glucosidase, leading to severe cardiac …

Fabry disease (FD) is an X-linked lysosomal storage disorder (LSD) caused by mutations of
the a-galactosidase A gene. The lysosomal enzyme a-galactosidase A (GLA) mediates the …

Adeno-associated virus (AAV) is a replication-deficient parvovirus that is extensively used as
a gene therapy vector. CD8+ T-cell responses against the AAV capsid protein can, however …