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Lentiviral gene therapy for X-linked chronic granulomatous disease.
Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ; Net4CGD consortium. Kohn DB, et al. Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27. Nat Med. 2020. PMID: 31988463 Free PMC article. Clinical Trial.
Effect of Cannabidiol on Drop Seizures in the Lennox-Gastaut Syndrome.
Devinsky O, Patel AD, Cross JH, Villanueva V, Wirrell EC, Privitera M, Greenwood SM, Roberts C, Checketts D, VanLandingham KE, Zuberi SM; GWPCARE3 Study Group. Devinsky O, et al. N Engl J Med. 2018 May 17;378(20):1888-1897. doi: 10.1056/NEJMoa1714631. N Engl J Med. 2018. PMID: 29768152 Free article. Clinical Trial.
Perspectives of Sickle Cell Disease Stakeholders on Heritable Genome Editing.
Hollister BM, Gatter MC, Abdallah KE, Armsby AJ, Buscetta AJ, Byeon YJJ, Cooper KE, Desine S, Persaud A, Ormond KE, Bonham VL. Hollister BM, et al. CRISPR J. 2019 Dec;2(6):441-449. doi: 10.1089/crispr.2019.0034. Epub 2019 Nov 19. CRISPR J. 2019. PMID: 31742431 Free PMC article.