Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

The move from reading to writing the human genome offers new opportunities to improve
human health. The United States National Institutes of Health (NIH) Somatic Cell Genome …

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 …

Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …

Abstract Adeno-associated virus (AAV)-mediated CRISPR-Cas9 editing holds promise to
treat many diseases. The immune response to bacterial-derived Cas9 has been speculated …

At present, the idea of genome modification has revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene …

Recent advances in gene editing technologies are enabling the potential correction of
devastating monogenic disorders through elimination of underlying genetic mutations …

Recent advances in base editing have created an exciting opportunity to precisely correct
disease-causing mutations. However, the large size of base editors and their inherited off …

Gene therapy has entered a new era after decades-long efforts, where the recombinant
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …