A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

IDOR-1117-2520 is now in a Phase 2 proof-of-concept trial in psoriasis, with the potential to expand into additional autoimmune conditions where the CCR6-CCL20 pathway plays a pathogenic role.

A new CRISPR breakthrough shows scientists can turn genes back on without cutting DNA, by removing chemical tags that act ...

This study offers important insight into the pathogenic basis of intragenic frameshift deletions in the carboxy-terminal domain of MECP2, which account for some Rett syndrome cases, yet similar ...

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 ...

A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...

Human gene maps contain major blind spots because they were built largely from the DNA sequences of people with European ancestry, according to a study published in Nature Communications. Researchers ...

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 ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...

A rare disease that has cut off children from the outside world now potentially has a cure. A clinical trial to treat severe combined immunodeficiency disorder (SCID), commonly referred to as “bubble ...

The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...

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 ...