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Page 1
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. Among authors: schefter le. Sci Transl Med. 2019 Jul 31;11(503):eaaw3768. doi: 10.1126/scitranslmed.aaw3768. Sci Transl Med. 2019. PMID: 31366580 Free PMC article.
Autologous, Gene-Modified Hematopoietic Stem and Progenitor Cells Repopulate the Central Nervous System with Distinct Clonal Variants.
Peterson CW, Adair JE, Wohlfahrt ME, Deleage C, Radtke S, Rust B, Norman KK, Norgaard ZK, Schefter LE, Sghia-Hughes GM, Repetto A, Baldessari A, Murnane RD, Estes JD, Kiem HP. Peterson CW, et al. Among authors: schefter le. Stem Cell Reports. 2019 Jul 9;13(1):91-104. doi: 10.1016/j.stemcr.2019.05.016. Epub 2019 Jun 13. Stem Cell Reports. 2019. PMID: 31204301 Free PMC article.
Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes.
Ozog S, Timberlake ND, Hermann K, Garijo O, Haworth KG, Shi G, Glinkerman CM, Schefter LE, D'Souza S, Simpson E, Sghia-Hughes G, Carillo RR, Boger DL, Kiem HP, Slukvin I, Ryu BY, Sorrentino BP, Adair JE, Snyder SA, Compton AA, Torbett BE. Ozog S, et al. Among authors: schefter le. Blood. 2019 Oct 17;134(16):1298-1311. doi: 10.1182/blood.2019000040. Blood. 2019. PMID: 31416800 Free PMC article.