Researchers at Mass General Brigham have developed a promising nasal spray for gene therapy, aiming to deliver treatments directly to the lungs. This innovation could offer a less invasive and more effective approach for genetic lung diseases like cystic fibrosis.
Gene Therapy
By inserting a complete gene, the technique provides a one-step solution that overcomes the limitations of CRISPR gene editing technology.
Rare yet debilitating disorders, Dravet syndrome, and other developing epileptic encephalopathies induce a variety of symptoms in children, such as seizures, intellectual incapacity, and even unexpected death. The majority of cases are brought on by a genetic mutation; variations in the sodium channel gene SCN1A are most frequently the cause […]
The outcomes of an international Phase III clinical trial conducted by researchers from the University of Pennsylvania Perelman School of Medicine and a multicenter group of investigators, published in the New England Journal of Medicine, show that adults with haemophilia B experienced an average 71% reduction in the number of […]
A Phase 1/2 trial included 15 individuals in total, among them three pediatric patients. The patients all suffered from the disease Leber congenital amaurosis caused by a gene that is essential in making proteins crucial to vision.
The protein dystrophin, absent in people with muscular dystrophy, will subsequently begin to be produced by the additional genetic code.
Researchers at the University of Iowa, the Broad Institute of MIT, Harvard, and Harvard have now created a gene-editing method that effectively fixes the most prevalent mutation affecting 85% of patients
Delivering a healthy copy of a gene to a person whose gene(s) are malfunctioning is known as gene therapy.
