StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

The heterogeneous nature of DBA mutations makes developing a single therapy challenging. Moreover, current gene therapy ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

Romance is a complex affair in humans. There’s personality, appearance, seduction, all manner of physical and social cues.

Lisa Bancroft is among thousands of people who have been tested for the faulty BRCA gene since its discovery 30 years ago.

People inherit two copies of each gene -; one from each parent -; an evolutionary fail-safe to ensure survival even when one of them doesn't function.

In a phase 2 trial, a gene therapy reduced angioedema attacks, which could significantly change the quality of life for ...

Under this two-hit model, Brugge says, one normal copy of BRCA1 ought to be able to carry out DNA repair even when the other ...

These preclinical findings could lead ... Single-Dose Gene Therapy Is Potentially Life-Changing for Adults With Hemophilia B Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding ...

DENVER, Nov. 6, 2024 /PRNewswire/ -- CS Genetics, a leader in next-generation single-cell genomics, today announced the ...

The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...