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 …

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 …

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

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 …

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 viral (AAV) vectors have emerged as the leading gene delivery platform
for gene therapy and vaccination. Three AAV-based gene therapy drugs, Glybera …

Preexisting humoral immunity to recombinant adeno-associated virus (AAV) vectors restricts
the treatable patient population and efficacy of human gene therapies. Approaches to clear …

Preexisting neutralizing antibodies (NAbs) against adeno-associated viruses (AAVs) pose a
major, unresolved challenge that restricts patient enrollment in gene therapy clinical trials …

Gene therapy is the straightforward approach for the application of recent advances in
molecular biology into clinical practice. One of the major obstacles in the development of …

Emerging coronaviruses (CoV) are constant global public health threats to society. Multiple
ongoing clinical trials for vaccines and antivirals against CoVs showcase the availability of …