Discarded Bone Marrow for Hematology Research
St. Jude Children's Research Hospital
Summary
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.
Description
The hemoglobinopathies (e.g. sickle cell disease (SCD) and thalassemia) are devastating inherited anemias that shorten and reduce quality of life. The only current curative therapy for SCD is bone marrow transplantation. However, many patients lack access to suitable donors for transplant. Alternative treatments based on gene therapy, gene editing and novel drugs are currently being developed and show great promise for hemoglobinopathies. Gene therapy and gene editing are especially appealing because they eliminate both the need for donors and the potentially devastating side effects of Graft-…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Patients of any age receiving orthopedic surgery for clinical management that involve bone marrow containing bone discard. * Patients receiving orthopedic surgery for clinical management will be considered for this study if they have the following diagnosis and criteria: * Homozygous S/S disease or doubly heterozygous for S and β thalassemia who are two years or older are eligible. * HbE-β- thalassemia or homozygous (severe) β-thalassemia. including those who are transfusion dependent (major) or severely anemic but relatively transfusion independent (intermedia). Di…
Location
- St. Jude Children's Research HospitalMemphis, Tennessee