Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Ocugen, Inc. (NASDAQ: OCGN) will present its latest advances in gene therapy for blindness diseases at NobleCon21, the annual ...

Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. Roche described the restructuring in its ...

A new FDA pathway could speed bespoke gene therapies, but key questions over scope and commercial viability remain.

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...

In preclinical work recently published in Nature Communications, Fred Hutch Cancer Center scientists used a genetic “chain reaction” to transform herpes simplex virus DNA during an HSV infection. The ...