Like base editing, prime editing offers a safer way to genome editing by relying on a nickase enzyme that “nicks” one DNA strand at a time, rather than cutting both simultaneously. Then, with the ...

Researchers at the Broad Institute and The Jackson Laboratory have used prime editing, a precise and versatile form of gene editing, to correct the root cause of AHC in mice. The team used a scalable ...

Alternating hemiplegia of childhood (AHC) is a rare, one-in-a-million genetic disease that affects kids. Most cases are caused by mutations in the ATP1A3 gene. That gene makes an ATPase enzyme that is ...

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 ...

Clustered, regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (CRISPR-Cas9) are genome editing tools. 1 After their potential in therapeutics was recognized, ...

For babies born with alternative hemiplegia of childhood (AHC), an extremely rare and severe neurodevelopmental disorder, there may be no obvious symptoms for several months. Then the attacks begin: ...

A variety of genetic diseases are caused by ‘nonsense’ mutations that change a sense codon to a premature termination codon (PTC), causing early termination of protein synthesis. Nonsense suppression, ...

Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder ...

A new paper from the laboratory of David Liu, PhD, at the Broad Institute describes a genome-editing strategy that could result in a one-time treatment for multiple unrelated genetic diseases. The new ...