Jorvet bone marrow manual

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Although sickle cell anemia was the first hereditary disease to be understood at the molecular level, there is still no adequate long-term treatment.

Characterization of the Role of the Pathogenicity Island and vapG

Allogeneic bone marrow transplantation is the only available cure, but this procedure is limited to a minority of patients with an available, histocompatible donor.

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Autologous transplantation of bone marrow stem cells that are transduced with a stably expressed, antisickling globin gene would benefit a majority of patients with sickle cell disease.

Antiapoptotic BCL-2 is required for maintenance of a model

Therefore, the development of a gene therapy protocol that corrects the disease in an animal model and is directly translatable to human patients is critical.

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