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 viruses (AAVs) are the safest and most effective gene delivery vehicles to
drive long-term transgene expression in gene therapy. While animal studies have shown …

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 …

The CRISPR-Cas9 system has raised hopes for developing personalized gene therapies for
complex diseases. Its application for genetic and epigenetic therapies in humans raises …

Abstract Adeno-associated virus (AAV)-derived viral vectors are a promising platform for the
delivery of curative, life-changing therapies to a huge number of patients with monogenic …

Given the increasing number of gene transfer therapy studies either completed or underway,
there is growing attention to the importance of preexisting adaptive immunity to the viral …

Immune responses to protein and peptide drugs can alter or reduce their efficacy and may
be associated with adverse effects. While anti-drug antibodies (ADA) are a standard clinical …

Alzheimer's disease (AD) is a progressive degenerative neurological disorder affecting
nearly one in nine elderly people in the United States. Population studies have shown that …

Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have
achieved sustained clinical improvement after a single systemic injection of adeno …

Adeno-associated virus (AAV) has a single-stranded DNA genome encapsidated in a small
icosahedrally symmetric protein shell with 60 subunits. AAV is the leading delivery vector in …