Meghan McLean Poon Ph.D., Karen Potter Ph.D., Yuying You Ph.D. Cell and gene therapies represent a transformative frontier in modern medicine, offering potential cures for previously untreatable ...
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 ...
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 ...
Pancreatitis is a complex inflammatory disease driven by both genetic and environmental factors. It poses substantial ...
ASGCT–OTXL joint venture platform goes live with demo at ASGCT 2026 Annual Meeting and applications opening this month for qualified Asset listings BOSTON, May 14, 2026 /PRNewswire/ -- The American ...
Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra copy of chromosome 21. Down syndrome is also the most common genetic cause ...
Presented improved editing data in non-human primates (NHP) for sickle cell disease (SCD), demonstrating a single dose of Gene Writer achieved an ...
Please provide your email address to receive an email when new articles are posted on . SB-007 is designed to treat address the root genetic cause of Stargardt disease. A phase 1/2 study investigating ...
A planned first-in-human trial aims to treat GM1 gangliosidosis in utero, possibly laying the foundation for prenatal gene ...
Please provide your email address to receive an email when new articles are posted on . The FDA granted investigational drug clearance to HG202, a CRISPR/Cas13Y RNA-editing therapy for neovascular age ...
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