Publications

@article{Bjurstrom2016Preservation,
title = {Preservation of Gene Edited Hematopoietic Stem Cells By Transient Overexpression of BCL-2 mRNA },
author = {Carmen Flores Bjurström and Michelle Mojadidi and Anastasia Lomova and Stephen Lai and Sorel Fitz-Gibbon and Matteo Pellegrini and Aaron R Cooper and Georgia R Lill and Michael Kaufman and Gregory J Cost and Michael C. Holmes and Roger P Hollis and Donald B Kohn},
doi = { https://doi.org/10.1182/blood.V128.22.3636.3636 },
volume = {128},
number = {22},
abstract = {Site-specific gene correction of the point mutation causing sickle cell disease (SCD) in hematopoietic stem cells (HSCs) constitutes a precise strategy to generate a life-long source of gene-corrected erythrocytes that do not sickle. However, low efficiency of homology-directed repair (HDR) in primitive reconstituting HSCs is currently a limit to the use of therapeutic genome editing for treatment of severe genetic blood disorders. To identify the mechanism(s) that underlie decreased HDR efficacy in primitive HSCs relative to that in more mature progenitor populations, we assessed: efficiency of gene delivery and expression after electroporation of in vitro transcribed mRNA; functional ZFN-mediated endonuclease activity; cell cycle status; gene expression of key HDR genes; and cytotoxic responses; in the following immunophenotypically-defined human cell populations: HSCs (CD34+/CD38-/CD90+CD45RA-); multipotent progenitors (MPPs) (CD34+/CD38-/CD45RA-/CD90-); and progenitor cells (CD34+/CD38+).},
keywords = {bcl2 gene, cd34 antigens, cytotoxicity, electroporation, genes, genome editing, hematopoietic stem cells, messenger, oligonucleotides, protein overexpression, rna},
pubstate = {published},
tppubtype = {article}
}