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 ...

Spinal muscular atrophy (SMA) is a devastating pediatric neuromuscular disorder caused by loss-of-function mutations in the SMN1 gene, which prevents the body from producing enough of the survival ...

We are often confronted with a simple question, “which gene editing technique is the best?”; the simple answer is “there isn’t one”. In 2021, a year after prime editing first made its mark, we ...

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 ...

Gretchen Gulla is a strategy editor at The Wall Street Journal, focused on National Affairs coverage.

Use validated, sequence-verified nucleases, guides, and donors to reduce run-to-run variability and simplify comparability as indications expand. Layer the Analytics Combine nanoparticle ...