The move aims to regulate cell and gene therapies, and xenografts or animal-to-human transplants. Such therapies are ...
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 ...
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.
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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