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 ...

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 ...

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...

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 ...

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 ...

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 ...

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 ...

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 ...

Researchers have developed a gene-editing treatment for prion disease that extends lifespan by about 50 percent in a mouse model of the fatal neurodegenerative condition. The treatment, which uses ...