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Bone Marrow Sampling and Transplants

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Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
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Disorders of erythrocytes, or red blood cells (RBCs), include a range of conditions affecting their number, shape, or function.
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Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
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Toward life without sickle cell disease

Utpal P Davé1, Jörg Bungert2

  • 1Department of Medicine, Division of Hematology/Oncology, Indiana University School of Medicine, Roudebush VA Medical Center, and IU Simon Comprehensive Cancer Center, Indianapolis, IN, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|September 18, 2024
PubMed
Summary

No abstract available in PubMed .

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