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TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction.
Lux CT, Pattabhi S, Berger M, Nourigat C, Flowers DA, Negre O, Humbert O, Yang JG, Lee C, Jacoby K, Bernstein I, Kiem HP, Scharenberg A, Rawlings DJ. Lux CT, et al. Mol Ther Methods Clin Dev. 2018 Dec 31;12:175-183. doi: 10.1016/j.omtm.2018.12.008. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30705922 Free PMC article.
Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.
Humbert O, Radtke S, Samuelson C, Carrillo RR, Perez AM, Reddy SS, Lux C, Pattabhi S, Schefter LE, Negre O, Lee CM, Bao G, Adair JE, Peterson CW, Rawlings DJ, Scharenberg AM, Kiem HP. Humbert O, et al. Sci Transl Med. 2019 Jul 31;11(503):eaaw3768. doi: 10.1126/scitranslmed.aaw3768. Sci Transl Med. 2019. PMID: 31366580 Free PMC article.
Therapeutic Gene Editing Safety and Specificity.
Lux CT, Scharenberg AM. Lux CT, et al. Hematol Oncol Clin North Am. 2017 Oct;31(5):787-795. doi: 10.1016/j.hoc.2017.05.002. Epub 2017 Jun 29. Hematol Oncol Clin North Am. 2017. PMID: 28895847 Free PMC article. Review.