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Conditions: Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, ADA SCID, XSCID
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 690 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2028
U.S. locations: 39
States / cities: Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 34 more

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions: Human Placental Derived Stem Cell; Drug
Lead sponsor: New York Medical College; Other
Eligibility: 0 Years to 55 Years
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2022
U.S. locations: 3
States / cities: Denver, Colorado • Valhalla, New York • Salt Lake City, Utah

Conditions: Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions: Hematopoietic stem cell transplant (HSCT); Biological
Lead sponsor: Paul Szabolcs; Other
Eligibility: 5 Years to 40 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Severe Combined Immunodeficiency, X Linked, Gene Therapy
Interventions: autologous CD34+ cell transduced with G2SCID vector; Biological
Lead sponsor: David Williams; Other
Eligibility: 0 Years to 5 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2028
U.S. locations: 4
States / cities: Los Angeles, California • Atlanta, Georgia • Boston, Massachusetts + 1 more

Conditions: Severe Combined Immunodeficiency
Interventions: Gene-Transduced Autologous CD34+ Stem Cells; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Months to 20 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2011
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions: anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation; Drug · Procedure
Lead sponsor: Fairview University Medical Center; Other
Eligibility: Up to 35 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2002
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 3,500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2005
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 55 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence
Interventions: Increlex; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years to 20 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Severe Combined Immunodeficiency
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 13 Years to 17 Years · Female only
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper IgE Syndromes, Hyper IgM Deficiencies, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, Common Variable Immune Deficiency (CVID)
Interventions: CD3/CD19 negative allogeneic hematopoietic stem cells; Biological
Lead sponsor: Paul Szabolcs; Other
Eligibility: 5 Years to 45 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Immunologic Deficiency Syndrome, Agammaglobulinemia, Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome, Common Variable Immunodeficiency
Interventions: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water; Drug
Lead sponsor: Grifols Therapeutics LLC; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2004
U.S. locations: 9
States / cities: Birmingham, Alabama • Denver, Colorado • Washington D.C., District of Columbia + 6 more

Conditions: Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: RTI International; Other
Eligibility: 1 Day to 31 Days
Enrollment: 30,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: X-Linked Combined Immunodeficiency Diseases
Interventions: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan; Biological · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years to 40 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2022
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Adenosine Deaminase Deficiency, Autosomal Recessive Disorder, Immune System Disorder, Purine-Nucleoside Phosphorylase Deficiency, Severe Combined Immunodeficiency, Severe Combined Immunodeficiency With Absence of T and B Cells, X-Linked Severe Combined Immunodeficiency
Interventions: Allogeneic Bone Marrow Transplantation, Cyclosporine, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation; Procedure · Drug · Other + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Not listed
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2018
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: X-linked Severe Combined Immunodeficiency (XSCID)
Interventions: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin; Biological · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years to 50 Years · Male only
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2032
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
Interventions: Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan, Stem Cell Transplantation, Fludarabine phosphate 40 mg, Melphalan, Alemtuzumab 0.2 mg, Fludarabine phosphate 30 mg, MESNA; Drug · Biological
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 50 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2026
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Severe Combined Immunodeficiency
Interventions: Gene transfer; Biological
Lead sponsor: David Williams; Other
Eligibility: Male only
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2023
U.S. locations: 3
States / cities: Los Angeles, California • Boston, Massachusetts • Cincinnati, Ohio

Conditions: X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
Interventions: Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells; Genetic · Drug · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 3 Years to 99 Years · Male only
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2034
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Severe Combined Immunodeficiency Disease, X-linked
Interventions: CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs; Genetic · Drug · Device
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 24 Months · Male only
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2034
U.S. locations: 3
States / cities: San Francisco, California • Memphis, Tennessee • Seattle, Washington

Conditions: Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions: BPX-501 and Rimiducid; Biological
Lead sponsor: Bellicum Pharmaceuticals; Industry
Eligibility: 4 Months to 55 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Immune System Diseases
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: Not listed
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
Interventions: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: Up to 21 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2026
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Hematologic Malignancies
Interventions: UCB and HPDSC; Procedure
Lead sponsor: Celgene Corporation; Industry
Eligibility: Up to 55 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2013
U.S. locations: 1
States / cities: New Orleans, Louisiana

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Natural History Study of SCID Disorders

Human Placental-Derived Stem Cell Transplantation

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

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

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

Evaluation of Patients With Immune Function Abnormalities

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1

Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients

Early Check: Expanded Screening in Newborns

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

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

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

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector

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

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

Developing Newborn Screening for Infants With Primary Immunodeficiency

Immune Disorder HSCT Protocol

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders