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Discarded Bone Marrow for Hematology Research

St. Jude Children's Research Hospital · 2022
ClinicalTrials.gov · Datasets · License: Public Domain · 2022
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The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals. The processing of samples will help to understand how best to manipulate HSPC's from hemoglobinopathy patients with gene therapy and gene technologies in the laboratory environment. It will also allow us to establish a reservoir of samples that can be studied in the future to assess cellular function and fitness for transplant. Secondary objectives * To develop gene transfer and gene editing strategies as potentially curative therapy for hemoglobinopathies (e.g. sickle cell disease (SCD) and β-thalassemia). * To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for hemoglobinopathies. * To examine the biology of bone marrow cells isolated from patients with hemoglobinopathies.
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