Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …

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 …

Replacing or editing disease-causing mutations holds great promise for treating many
human diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has …

Modern experimental technologies can assay large numbers of biological sequences, but
engineered protein libraries rarely exceed the sequence diversity of natural protein families …

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 …

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 …

Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the
path for a broader second wave of therapies and laid the foundation for next-generation …

Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …

Recombinant Adeno-associated virus (rAAV) is one of the main delivery vectors for gene
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …

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 …