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 ...
(L to R) First author Anoop Babu Vasandan, PhD, St. Jude Department of Immunology, co-corresponding author Caitlin Zebley, MD, PhD, St. Department of Bone Marrow Transplantation & Cellular Therapy and ...
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 ...
Early results from a study of newborn screening methods show that DNA analysis picks up many more preventable or treatable serious health conditions than standard newborn screening and is favored by ...
The US Food and Drug Administration (FDA) halted 27 gene therapy trials Tuesday following indications that a second child is showing signs of leukemia after treatment for X-linked Severe Combined ...
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 ...
The tally is grim: three high-profile cancer cases, including one death, in French boys who underwent pioneering gene therapy for X-linked severe combined immunodeficiency (X-SCID). Yet Edward ...
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