Gene therapy has become a clinical reality as market-approved advanced therapy medicinal
products for the treatment of distinct monogenetic diseases and B-cell malignancies. This …

Gene therapies are designed to address the root cause of disease. As scientific
understanding of disease prevention, diagnosis, and treatment improves in tandem with …

Huntington's disease (HD) is a dominant neurological disorder caused by an expanded HTT
exon 1 CAG repeat that lengthens huntingtin's polyglutamine tract. Lowering mutant …

Prime editors have a broad range of potential research and clinical applications. However,
methods to delineate their genome-wide editing activities have generally relied on indirect …

Cas9 can cleave DNA in both blunt and staggered configurations, resulting in distinct editing
outcomes, but what dictates the type of Cas9 incisions is largely unknown. In this study, we …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Undesired on-and off-target effects of CRISPR-Cas nucleases remain a challenge in
genome editing. While the use of Cas9 nickases has been shown to minimize off-target …

Spinal muscular atrophy (SMA) is caused by mutations in SMN1. SMN2 is a paralogous
gene with a C• G-to-T• A transition in exon 7, which causes this exon to be skipped in most …

Over the last decade, CRISPR has revolutionized drug development due to its potential to
cure genetic diseases that currently do not have any treatment. CRISPR was adapted from …

CRISPR/Cas systems have revolutionized biology and medicine, and have led to new
paradigms in disease diagnostics and therapeutics. However, these complexes suffer from …