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 ...

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 ...

Gene therapy is superior to haploidentical hematopoietic stem cell transplantation (HSCT) to treat X-linked severe combined immunodeficiency (SCID-X1), according to a report published online April 13 ...

French researchers say that four boys stricken with a usually fatal type of immune deficiency are living normal lives after gene therapy procedures done, in one case, as long ago as 2 1/2 years ago. A ...

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 ...