While researchers have long appreciated the enormous potential of genome editing to treat disease, applications of these technologies in humans have historically been limited by safety concerns. Even ...

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 ...

Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base ...

Researchers at ETH Zurich in Basel report they have used CRISPR-Cas technology to decipher how mutations in a cell’s genome affect its function. With their new approach, the researchers can generate ...

Inborn errors of metabolism (IEMs) are rare, devastating disorders caused by pathogenic variants in genes encoding key metabolic enzymes, with the liver playing a critical role in over 140 IEMs. Liver ...

New gene editing techniques, like adenine base editing and prime editing, are creating new possibilities for treating dilated cardiomyopathy, a heart condition affecting about 1 in 250 people ...

Discover how prime editing is redefining the future of medicine by offering highly precise, safe, and versatile DNA corrections, bringing hope for more effective treatments for genetic diseases while ...

Prime editing, a versatile form of gene editing that can correct most known disease-causing genetic mutations, now has a new vehicle to deliver its machinery into cells in living animals. A team of ...

Scientists at the Broad Institute of MIT and Harvard have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently ...

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 ...