The emergency use authorizations (EUAs) of two mRNA-based severe acute respiratory
syndrome coronavirus (SARS-CoV)-2 vaccines approximately 11 months after publication of …

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 …

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 …

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 …

Hemophilia is a hereditary disease that remains incurable. Although innovative treatments
such as gene therapy or bispecific antibody therapy have been introduced, substantial …

Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

Many genetic diseases and undesirable traits are due to base-pair alterations in genomic
DNA. Base-editing, the newest evolution of clustered regularly interspaced short palindromic …

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

Because of their small size, the recently developed CRISPR-Cas12f nucleases can be
effectively packaged into adeno-associated viruses for gene therapy. However, a systematic …

On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …