A new study describes a base editing method for treating spinal muscular atrophy (SMA). This supports early signs that “single-shot” treatments from clinical trials using genome editing technologies ...

One small atom can make a big difference in a molecule’s pharmacological properties. Especially if that atom is a nitrogen. Chemists at the University of Oklahoma have now unveiled a new strategy for ...

Researchers at the Broad Institute of MIT and Harvard have developed a gene-editing treatment for prion disease that extends ...

Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein. The combination of gene editing and patient-specific stem cells may offer a new ray of ...

Researchers have developed a gene editing strategy that could repair the genetic problems that lead to Huntington's disease and Friedreich's ataxia. These disorders arise because of abnormal repeats ...

A team of researchers at the Broad Institute, led by gene-editing pioneer David Liu, has developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple ...

Spinal muscular atrophy is a leading genetic cause of infant death worldwide, and is typically caused by loss-of-function mutations in the SMN1 gene, which produces the SMN protein Gene therapy and ...

While CRISPR-mediated gene editing has led to powerful advances across biology, medicine, and agriculture, challenges persist in optimizing the editing efficiency of enzymes, such as the widely used ...

CAMBRIDGE, Mass. & MENLO PARK, Calif.--(BUSINESS WIRE)--Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based ...