A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Key takeaways. Aging is not an inevitable process and can potentially be reversed. People may live into the twenty-second ...

Officials at Caring Cross emphasize that the organization is dedicated to ensuring the global affordability of advanced therapies.

High school teacher Marcus Mitchell, 35, is the first adult at Duke University Hospital to undergo this gene therapy aimed at curing sickle cell disease.

The rare pediatric disease affects approximately one in one million children globally. Mutations in the ITGB2 gene disrupt ...

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

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine This voice experience is generated by AI. Learn more. This ...

A new gene therapy, Celosia's CTx1000, is now being tested for ALS in a small clinical trial in Australia. The one-time treatment targets toxic TDP-43 protein clumps, a key driver of most cases of ALS ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit.  | Rocket ...