Clinical Trials

Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

cliniMACs HUD for T Cell Depletion

Autologous Gene Therapy for Artemis-Deficient SCID

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Evaluation of Patients With Immune Function Abnormalities

Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

Genetic Basis of Immunodeficiency

Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection

The Experience of Screening for SCID

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Study to Evaluate the Safety and Efficacy of USL261 (Intranasal Midazolam) in Patients With Seizure Clusters