2025年12月7日，《New England Journal of Medicine》发表了一项里程碑式研究，题为"Prime Editing for p47phox-Deficient Chronic Granulomatous Disease"。研究团队开发了PM359——一种基于Prime Editing技术的自体CD34+造血干细胞疗法，能够精准纠正NCF1基因中的两核苷酸缺失 ...

Researchers at the Wellcome Sanger Institute used prime editing to develop a novel tool to predict the chances of successfully inserting a gene-edited sequence of DNA into the genome of a cell. An ...

Prime Medicine is a gene editing company at the forefront of genetic therapies, with a promising pipeline and preclinical successes. The company has made substantial investments in R&D, demonstrating ...

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 ...

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 ...

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 ...

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) ...

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 ...

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 ...