X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder characterized by high susceptibility to infections that results from genetic mutations in the cytochrome b-245 beta chain ...

Scientists at St. Jude Children’s Research Hospital report that they have developed an unbiased, sensitive, and resource-efficient method to identify small, off-target sites that pose a safety risk ...

The trajectory of base editing has been remarkable, progressing from the laboratory to patient care, treating debilitating or terminal illnesses, in less than a decade. A type of gene editing that ...

A schematic showing the cytosine base editing intermediate and the various outcomes that can occur when the UNG protein is active. You may have seen it in the news recently: a baby in Pennsylvania ...

A collaborative effort between investigators at the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) and Massachusetts General Hospital (MGH), a founding ...

TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...