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Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ HSPCs Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral bl…
Evaluating a Clinical Decision Support Tool for Antiretroviral Therapy Optimization
This study is testing software designed to help healthcare providers choose the best HIV treatment combinations for their patients. HIV medicines, known as ant…
A Pilot Study in the Treatment of Refractory Epstein-Barr Virus (EBV) Infection With Related Donor EBV Cytotoxic T-Lymphocytes in Children, Adolescents and Young Adult Recipients
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be admin…
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and po…
A Pilot Study in the Treatment of Refractory Adenovirus (ADV) Infection With Related Donor ADV Cytotoxic T-Lymphocytes (ADV-CTLs)in Children, Adolescents and Young Adult Recipients
Related donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered …
Screening and Baseline Assessment of Patients With Abnormalities of Immune Function
This study will evaluate patients with abnormal immune function that results in recurrent or unusual infections or chronic inflammation. This may include inher…
Biologics And Clinical Immunology Cohort at Sinai
The study team plans to establish a bioregistry of patients receiving biologic therapy as part of their standard treatment at the Mount Sinai Therapeutic Infus…
The Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform Program (SDS-GPS Program)
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in t…
Natural History Study for Genetic Diseases of Allergic Inflammation and Immune Dysregulation
This protocol is a natural history study designed to evaluate subjects (and some family members) with suspected or identified genetic diseases of allergic infl…
Single Center Phase I Study of Adoptive Immunotherapy of Refractory Viral Infection With ex Vivo Expanded Rapidly Generated Virus Specific T (R-MVST) Cells for Immunodeficient Children and Young Adults
The primary objective is to determine the safety and feasibility of administering R-MVST cells to patients with refractory viral reactivation and/or symptomati…
Umbilical Cord Blood Transplantation From Unrelated Donors
This study is being done to determine how long it takes for the engraftment (recovery of blood cell counts) of umbilical cord stem cells and also how often eng…
Screening Protocol for Genetic Diseases of Lymphocyte Homeostasis and Programmed Cell Death
This study will determine the biochemical and genetic causes of inherited immune diseases affecting lymphocyte homeostasis. Lymphocytes are a type of white blo…