Sentences with phrase «x-linked severe combined immunodeficiency»
The LVSCID clinical trial for X-linked severe combined immunodeficiency uses a lentiviral vector to insert a healthy copy of the IL2RG gene into blood cells.
https://www.stjude.org/
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.
Read about the St. Jude trial: LVXSCID - ND: Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
St. Jude has played a key role in vector development, pioneering innovative vector designs for patients with hemophilia and the devastating immune disorder X-linked severe combined immunodeficiency (SCID).
This was a virus - mediated therapeutic gene to treat X-linked severe combined immunodeficiency.
Fischer, in 2000, reported demonstrating the clinical efficacy of gene therapy for the first time, using blood stem cells to treat a fatal genetic disorder called X-linked severe combined immunodeficiency.
Not exact matches
Severe combined immunodeficiency — X1 (SCID - X1) is an X-linked inherited disorder characterized by an early block in T and natural killer (NK) lymphocyte differentiation.