In vivo base editing enables precise, single-nucleotide changes to DNA without double-strand breaks. A recent application in ...

We spoke with the developers and manufacturers of the CRISPR gene therapy used to treat KJ Muldoon’s rare genetic condition, CPS1, about how the product was created in just six months.

A Ph.D. student in biomolecular engineering at the University of California, Santa Cruz, has built a software program ...

CRISPR is revolutionizing medicine by enabling precise DNA edits, treating genetic diseases like sickle cell anemia and ...

Emerging gene therapies-such as the recent, promising individualized CRISPR gene editing treatment of a baby with a severe UCD called CPS1 deficiency-are both raising hope among UCD patients and ...

Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with genetic diseases?

A Californian baby was diagnosed shortly after birth with CPS1 deficiency—a rare, life-threatening metabolic disorder. This case became the world's first personalized CRISPR gene-editing therapy ...

The infant was born with a metabolic disorder called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, in which the lack of an enzyme causes a build-up of ammonia in the blood, leading to ...

This innovative software, CRISPRware, optimizes guide RNA design for genome editing, supporting research on genetic disorders ...