The move aims to regulate cell and gene therapies, and xenografts or animal-to-human transplants. Such therapies are ...
A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...
Primary plasma cell leukemia (pPCL) is a rare and highly aggressive plasma cell malignancy, often associated with complex ...
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Gene Therapy Shows Lasting Benefit for Children With Rare Disorder
For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...
Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy.
New research has identified markers of two critical stem cell populations that drive colorectal cancer formation.
As of a September 25, 2025 data cut-off-date, 10 patients with treatment-refractory, moderate-to-severe Systemic Lupus Erythematosus (SLE) were treated with a single dose of FT819 with less-intensive ...
New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial ...
Key takeawaysA blood stem cell gene therapy co-developed by UCLA's Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare ...
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