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Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, Bauer DE. Wu Y, et al. Nat Med. 2019 May;25(5):776-783. doi: 10.1038/s41591-019-0401-y. Epub 2019 Mar 25. Nat Med. 2019. PMID: 30911135 Free PMC article.
Evolving Gene Therapy in Primary Immunodeficiency.
Thrasher AJ, Williams DA. Thrasher AJ, et al. Mol Ther. 2017 May 3;25(5):1132-1141. doi: 10.1016/j.ymthe.2017.03.018. Epub 2017 Mar 31. Mol Ther. 2017. PMID: 28366768 Free PMC article. Review.
Emerging Genetic Therapy for Sickle Cell Disease.
Orkin SH, Bauer DE. Orkin SH, et al. Annu Rev Med. 2019 Jan 27;70:257-271. doi: 10.1146/annurev-med-041817-125507. Epub 2018 Oct 24. Annu Rev Med. 2019. PMID: 30355263 Review.
Emerging cellular and gene therapies for congenital anemias.
Ludwig LS, Khajuria RK, Sankaran VG. Ludwig LS, et al. Am J Med Genet C Semin Med Genet. 2016 Dec;172(4):332-348. doi: 10.1002/ajmg.c.31529. Epub 2016 Oct 28. Am J Med Genet C Semin Med Genet. 2016. PMID: 27792859 Free PMC article. Review.
Thrombopoietin: tickling the HSC's fancy.
Kim AR, Sankaran VG. Kim AR, et al. EMBO Mol Med. 2018 Jan;10(1):10-12. doi: 10.15252/emmm.201708450. EMBO Mol Med. 2018. PMID: 29191946 Free PMC article.
Control of human hemoglobin switching by LIN28B-mediated regulation of BCL11A translation.
Basak A, Munschauer M, Lareau CA, Montbleau KE, Ulirsch JC, Hartigan CR, Schenone M, Lian J, Wang Y, Huang Y, Wu X, Gehrke L, Rice CM, An X, Christou HA, Mohandas N, Carr SA, Chen JJ, Orkin SH, Lander ES, Sankaran VG. Basak A, et al. Nat Genet. 2020 Feb;52(2):138-145. doi: 10.1038/s41588-019-0568-7. Epub 2020 Jan 20. Nat Genet. 2020. PMID: 31959994 Free PMC article.
25 results