The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...
A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...
A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a unique window shortly after birth in which circulating blood stem cells can ...
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 ...
A boy with an ultra-rare immune disease which can carry a “death sentence” is living a normal life after taking part in a groundbreaking gene therapy trial. Four-year-old Eisa Hussain now enjoys ...
After 44 days, Kendric Cromer, 12, left the hospital. While his family feels fortunate that he was the first to receive a treatment, their difficult experiences hint at what others will be up against.
In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...
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