The prime editor is a fusion protein consisting of a catalytically impaired Cas9 nickase (Cas9n) and an engineered reverse transcriptase (RT). The Cas9n is guided by a prime editing guide RNA (pegRNA) ...

Prime Medicine’s prime editors have corrected a gene mutation in primates that causes a rare liver disease, a step toward taking the tech to humans. In an Oct. 27 presentation at the annual European ...

On track to file IND and/or CTA for PM577 in Wilson Disease in H1 2026 and for PM647 in AATD mid-2026; initial clinical data from both ...

Despite the copious demands of his administrative positions as the White House chief science advisor (and until last year, the director of the NIH), Francis Collins, MD, PhD, has continued to run a ...

While Kiran Musunuru, MD, PhD, is already in the process of using prime editing—a search and replace genome editing technology that doesn’t create double-strand breaks or use donor DNA—in his academic ...

New technology enables the insertion of a large segment of DNA into a genome, potentially expanding gene therapy treatment ...

image: Mitchell Weiss, M.D., Ph.D. and Jonathan Yen, Ph.D., of St. Jude Children's Research Hospital view more (MEMPHIS, Tenn. – April 17, 2023) Sickle cell disease (SCD) is a serious blood disorder ...

CRISPR-Cas9 gene editing relies on a guide RNA that binds to a desired DNA sequence and a Cas9 enzyme that cuts both strands of DNA at that site, creating a double-strand break. Scientists edit the ...

This study is led by Prof. Xianqun Fan (Department of Ophthalmology, Shanghai Jiao Tong University School of Medicine Affiliated Ninth People's Hospital). Gene editing ushers in a new era of disease ...

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 ...

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: ...

Scientists showed how prime editing can correct mutations that cause sickle cell disease in a potentially curative approach. Sickle cell disease (SCD) is a serious blood disorder affecting millions of ...