Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

CHICAGO, Nov 8 (Reuters) - A single infusion of CRISPR Therapeutics' (CRSP.BN), opens new tab experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half ...

This image shows three brain cells with the faulty protein that causes Huntington’s disease. The bright yellow cell in the middle has built up a clump of this protein inside it. The blue spots in the ...

UC San Diego scientists have created a gene therapy that goes beyond masking Alzheimer’s symptoms—it may actually restore brain function. In mice, the treatment protected memory and altered diseased ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

Intellia Therapeutics CEO John Leonard explained the circumstances of the patient death at the JP Morgan Healthcare ...

A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene therapy for ...