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 …

Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …

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 …

BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

There has been a resurgence in gene therapy efforts that is partly fueled by the identification
and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non …

In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

While there has been substantial progress and notable achievements in the use of adeno-
associated virus (AAV) gene therapy vectors for treatment of rare diseases, setbacks related …

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

The field of gene therapy has made considerable progress over the past several years.
Adeno-associated virus (AAV) vectors have emerged as promising and attractive tools for in …