No Image

World’s first gene therapy for glycogen storage disease produces remarkable results

September 30, 2019 dna 0
The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in the form of cornstarch every few hours for survival. If a cornstarch dose is missed, the disease can lead to seizures and even death. A clinical trial originally set out to simply test the safety and dosage of the gene therapy for three patients with GSD Type Ia. The dramatic improvement in their lives was unexpected.
No Image

Researchers developing new ‘DNA stitch’ to treat muscular dystrophy

September 30, 2019 dna 0
A new therapeutic being tested is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD). The treatment — a cocktail of DNA-like molecules — results in dramatic regrowth of a protein called dystrophin, which acts as a support beam to keep muscles strong. The protein is virtually absent in those with DMD.
No Image

New AI method may boost Crohn’s disease insight and improve treatment

September 30, 2019 dna 0
Scientists have developed a computer method that may help improve understanding and treatment of Crohn’s disease, which causes inflammation of the digestive tract. The study used artificial intelligence to examine genetic signatures of Crohn’s in 111 people. The method revealed previously undiscovered genes linked to the disease, and accurately predicted whether thousands of other people had the disease.