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 …

Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …

Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …

Adeno-associated virus (AAV) has emerged as a leading platform for gene delivery for
treating various diseases due to its excellent safety profile and efficient transduction to …

With the improvement of gene vectors, the rise of chimeric antigen receptor T cell
immunotherapy and breakthroughs in the genome editing technology, gene therapy had …

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 …

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 …

The optimization of upstream and downstream processes for production of recombinant
adeno-associated virus (rAAV) with consistent quality depends on the ability to rapidly …

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 …