Both CBEs and ABEs have been extensively engineered to improve their efficiency, specificity, and targeting range, enabling a wide variety of base pair conversions across the genome. Base editing can ...

A reasonable idea, suggests Sekar Kathiresan, MD, co-founder and CEO of Verve Therapeutics, is the development of a one-time treatment—a single spelling change in the DNA of a liver gene in an adult ...

David R. Liu, PhD, a professor at Harvard University, the Broad Institute of MIT and Harvard, and the Howard Hughes Medical Institute A research team led by gene editing pioneer David R. Liu, PhD, ...

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 ...

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 ...

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 ...

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 ...

I covered Beam Therapeutics (BEAM) in April last year, when the stock had a market cap of $3.7bn. Today, despite some derisking events, it has a market cap of $2.6bn. The stock price has fallen 30% as ...