A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with ...
Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentivira ...
Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening. MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders cha ...