Early research shows that editing expanding trinucleotide repeats halts the lengthening process that causes neurological ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

In 2012, scientists Jennifer Doudna and Emmanuelle Charpentier replicated this mechanism found in microbes to develop a ...

Researchers have used a personalised base-editing gene therapy to successfully treat an infant with carbamoyl-phosphate ...

The baby boy received the bespoke therapy within a few months of birth and is doing well, according to a presentation at ASGCT.

In a medical first that could revolutionize treatment for thousands of rare genetic diseases, doctors have successfully used ...

In addition to the five nitrogenous bases—adenine, guanine, cytosine, thymine and uracil—required for building DNA and RNA, the researchers also found xanthine, hypoxanthine, and nicotinic ...

New research demonstrates the potential of precise genome editing technologies, called adenine base editors, to correct disease-causing mutations in stem cells from patients with X-linked chronic ...

today presented a poster titled “Engineering of compact and efficient adenine base editors from metagenomic derived systems” at the American Institute of Chemical Engineers (AIChE) 7th ...

A one-time A-to-G correction of the PiZ mutation with Beam’s adenine base editor has the potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that causes toxicity ...