New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

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 ...

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 ...

Over the last 15 years, I have witnessed how cell and gene therapy (CGT) has evolved in many ways, spanning from research and development to manufacturing. Breakthroughs such as CAR-T and gene therapy ...

In a study of human immune cells infected with HIV, the virus that causes AIDS, scientists at Johns Hopkins Medicine say a molecule within HIV itself can be manipulated and amplified to force the ...

For stem cells to differentiate into the appropriate cell-type, transcription factors (TFs) must be tailored to highly specific expression patterns of lineage-specific genes. Abnormalities in the ...

Please provide your email address to receive an email when new articles are posted on . RGX-202 was safe and well-tolerated in all 12 patients at two different doses with no serious adverse events.

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 ...