Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in
SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads …

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 …

Due to the high prevalence and long incubation periods often without symptoms, the severe
acute respiratory syndrome coronavirus-2 (SARS-CoV-2) has infected millions of individuals …

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

Gene therapy is the product of man's quest to eliminate diseases. Gene therapy has three
facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where …

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 …

Gene therapies are currently one of the most investigated therapeutic modalities in both the
preclinical and clinical settings and have shown promise in treating a diverse spectrum of …

The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …