Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

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 is at an inflection point. Recent successes in genetic medicine have paved the
path for a broader second wave of therapies and laid the foundation for next-generation …

In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo
gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer …

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …

Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of
hexosaminidase A (HexA). Here, we describe an adeno-associated virus (AAV) gene …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

Two patients with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene
encoding superoxide dismutase 1 (SOD1) were treated with a single intrathecal infusion of …