Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

We treated a 27-year-old patient with Duchenne's muscular dystrophy (DMD) with
recombinant adeno-associated virus (rAAV) serotype 9 containing d Sa Cas9 (ie,“dead” …

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 …

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 …

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 …

The success of base editors for the study and treatment of genetic diseases depends on the
ability to deliver them in vivo to the relevant cell types. Delivery via adeno-associated viruses …

Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

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 …