About 15 years ago, David Liu, PhD, a young chemistry professor at Harvard University, launched an unusually profitable extracurricular activity. Every few months, he’d accompany a handful of students ...

Point mutations change one or very few nucleotides in DNA, but they cause an estimated 30,000 genetic diseases in humans. For example, children born with Progeria age rapidly and die before adulthood, ...

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 ...

At least five groups have independently invented a new class of CRISPR gene editing tools called dual base editors. The systems combine the functions and components of two CRISPR base editors, which ...

In a new approach to genome editing called “base editing,” researchers have engineered the CRISPR-Cas9 enzyme to modify individual DNA bases more efficiently and more accurately than with existing ...

Two new studies offer more validation that prime editing and base editing have the potential to permanently fix a gene variant associated with the rare disease phenylketonuria. In two separate papers ...

Adenosine base editing restarted fetal hemoglobin expression in cells from patients with sickle cell disease. Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease ...