Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

Gabriella Ngang, of Farmington Hills, is one of three in Michigan to take part in a clinical trial of a new sickle cell ...

The FDA approved the first gene-editing therapy using Nobel Prize-winning CRISPR technology on Dec. 8, 2023. | Sarah Silbiger/Getty Images By Lauren Gardner and Katherine Ellen Foley 12/08/2023 11 ...

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

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

UCD team uses novel polymer-based system to deliver corrective genes, an approach that appears to be better tolerated by ...

RNA editing, then, is also a type of gene therapy. Its goal is to change how RNA interprets genetic instructions to control how proteins are made. In most recent advancements, ...

A breakthrough treatment for sickle cell patients could soon become the first gene-editing treatment to be approved by the Food and Drug Administration (FDA). Sickle cell disease (SCD) is an ...

A man who was the first person in the UK to receive a groundbreaking gene-editing therapy on the NHS, said he felt " ...

Gene Therapy/Editing Series 1: A Brief Introduction To Gene Therapy Oct. 28, 2019 12:53 PM ET BLUE , GSK , ORTX , QURE , SGMO 5 Comments 7 Likes Bhavneesh Sharma ...

Gene therapy and editing are still in the experimental stage, and their long-term safety and efficacy are not fully understood. These treatments have the potential to revolutionize medicine and ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy.The treatment corrects ...