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Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus

• Source: Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and

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Characterization of Expanded Gamma Delta T Cells from Atypical X-SCID Patient Reveals Preserved Function and IL2RG-Mediated Signaling

• Authors : Tuovinen, EA; Poysti, S; Hamdan, F

Subjects: Medicin och hälsovetenskap

• Source: Journal of clinical immunology. 43(2):358-370

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JSP191 Antibody Targeting Conditioning in SCID Patients

• Source: A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of JSP191 for Hematopoietic Cell Transplantation Conditioning to Achieve Engraftment and Immune Reconstitution in Subjects With

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Autologous Mobilized Peripheral Blood CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Transduced With the Elongation Factor Alpha Short Promoter (EFS) - Adenosine Deaminase (ADA) Gene (EFS-ADA) Lentiviral Vector for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID)

• Authors : Satiro N De Oliveira, Assistant Professor of Pediatrics

• Source: Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K,

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Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID (RAG1-SCID)

• Authors : ZonMw: The Netherlands Organisation for Health Research and Development; Horizon 2020 - European Commission; alankester, Principal Investigator

• Source: Garcia-Perez L, van Eggermond M, van Roon L, Vloemans SA, Cordes M, Schambach A, Rothe M, Berghuis D, Lagresle-Peyrou C, Cavazzana M, Zhang F, Thrasher AJ, Salvatori D, Meij P, Villa A, Van Dongen

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Conditioning SCID Infants Diagnosed Early (CSIDE)

• Authors : Michael Pulsipher, MD, Sponsor-Investigator; Protocol Co-Chair

• Source: A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRαβ+/CD19+ Depleted Transplantation: A

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The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions.

• Authors : Pai, Sung-Yun; Griffith, Linda; Cuvelier, Geoffrey

Subjects: Omenn syndrome; SCID; Severe combined immunodeficiency

• Source: Journal of Allergy and Clinical Immunology. 151(2)

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Autologous Gene Therapy for Artemis-Deficient SCID

• Authors : Morton Cowan, Professor Emeritus

• Source: Buckley RH. The multiple causes of human SCID. J Clin Invest. 2004 Nov;114(10):1409-11. doi: 10.1172/JCI23571.Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA.

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

• Source: Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated With Strimvelis (Previously GSK2696273) Gene Therapy: Long-Term

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An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

• Authors : Great Ormond Street Hospital for Children NHS Foundation Trust; Donald B. Kohn, M.D., Principal Investigator

• Source: An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-

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