A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...

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 ...

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 ...

The American Society of Health-System Pharmacists has launched the  ASHP Center for Next Generation Therapeutics, aimed at supporting hospitals and ...

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 ...

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 ...