Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A.

ABSTRACT: A hallmark of Fanconi anemia is accelerated decline in hematopoietic stem and progenitor cells (CD34 +) leading to bone marrow failure. Long-term treatment requires hematopoietic cell transplantation from an unaffected donor but is associated with potentially severe side-effects. Gene therapy to correct the genetic defect in the patient's own CD34⁺ cells has been limited by low CD34⁺ cell numbers and viability. Here we demonstrate an altered ratio of CD34^Hi to CD34^Lo cells in Fanconi patients relative to healthy donors, with exclusive in vitro repopulating ability in only CD34^Hi cells, underscoring a need for novel strategies to preserve limited CD34⁺ cells. To address this need, we developed a clinical protocol to de

SUBMITTER: Adair JE 

PROVIDER: S-EPMC6278989 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature