The ability of protein kinases to transfer a phosphate group to target proteins plays an important role in many cellular ...

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

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

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

Baby KJ had been diagnosed with carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after his birth in August 2024. In a mere six months, scientists across academia and industry developed and ...

The patient was born with severe carbamoyl phosphate synthetase 1 deficiency. Standard treatment for the rare metabolic disease is a liver transplant, but the patient was not eligible for these ...

KJ Muldoon, a 10-month-old who was born with severe carbamoyl phosphate synthetase 1 deficiency, received the first infusion of the bespoke therapy in February.

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he was born. He got out of hospital on Tuesday.

Carbamoyl phosphate synthetase I (CPS1) is an enzyme that is crucial for breaking down the ammonia that is formed when proteins are broken down.

In a recent New England Journal of Medicine report, researchers describe a revolutionary gene editing technology that has been used to treat an infant with carbamoyl-phosphate synthetase 1 (CPS1 ...

Loading audio... Scientists took months to develop gene therapy for baby KJ Muldoon to treat his rare genetic disease. KJ from Clifton Heights, Pennsylvania, was born with a rare metabolic condition, ...