X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in T-cell, B-cell, and natural killer (NK) cell function, caused by mutations in ...
A gene therapy for a rare type of severe combined immunodeficiency (SCID) was safe and sufficiently restored T-cell immunity in an early trial, results from a phase I/II study showed. At a single ...
Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say. Experts said the findings are ...
Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life. Patients with ...
RMAT designation follows positive Phase 1/2 clinical data published in the New England Journal of Medicine Designation to facilitate expedited development and review of MB-107 for XSCID NEW YORK and ...
A new report says early evidence of using gene therapy to treat monogenic diseases offers reason for optimism. Gene therapy (GT) holds significant potential to help correct monogenic diseases, ...
Researchers from Nijmegen and Newcastle discovered a new genetic mutation leading to severe combined immune deficiency disorder (SCID). It’s the first time a mutation in the proteasome, a molecular ...
Current GT strategies employ viral vectors (eg, murine g-retroviruses or lentiviruses) to introduce genetic material into isolated HSCs. However, gene editing (eg, CRISPR-based GT) is under active ...
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