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J.E. Adair et al.
MOI suggest that the mixed cell culture supports trans- duction of hematopoietic progenitor cells, at least.
In conclusion, we describe an alternative strategy to a direct, immunomagnetic bead-based selection of CD34-expressing cells that overcomes current barriers in isolation of blood stem and progenitor cells especially for diseases like FA. Our novel approach to preserve available CD34+ cells during initial blood product processing has the potential to improve gene therapy and gene editing in set- tings of limited CD34+ cell availability, including FA and other diseases in which direct CD34 enrichment has proven inefficient, such as SCD.
Acknowledgments
We thank our patients and their families. We thank the Fanconi Anemia Research Fund for sponsoring patient travel and housing arrangements associated with this study. We especially thank the Fred Hutch Cell Processing Facility and Seattle Cancer Care Alliance for their support in enrollment and treatment of clinical trial patients. We thank H. Crawford and K. Gonzalez for help in preparing the manuscript. We
also thank J. Chen and C. Ironside for excellent support in our mouse studies. This work was primarily funded by a Sponsored Research Agreement between Fred Hutch and Rocket Pharmaceuticals.
Funding
This work was also supported in part by grants and contracts to H-PK from the NIH HHSN2680004 and HL122173 and by funds to JEA from the Fred Hutch. This research was also funded in part through the NIH/NCI Cancer Center Support Grant P30 CA015704. H-PK is a Markey Molecular Medicine Investigator and received support as the inaugural recipient of the José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research and the Fred Hutch Endowed Chair for Cell and Gene Therapy. JEA and H-PK are co-inventors on U.S. Provisional Patent Applications #62/491,116 and #62/503,801 “Novel Manufacturing of Gene Corrected Autologous Blood Cells for Gene Therapy.” JEA has previously served as a consultant for Rocket Pharmaceuticals. H-PK is an active consultant for Rocket Pharmaceuticals. The other authors declare that they have no competing interests.
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