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1,247 results

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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. Among authors: williams da. 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.
Rac GTPases in human diseases.
Pai SY, Kim C, Williams DA. Pai SY, et al. Among authors: williams da. Dis Markers. 2010;29(3-4):177-87. doi: 10.3233/DMA-2010-0738. Dis Markers. 2010. PMID: 21178276 Free PMC article. Review.
Out of harm's way.
Williams DA, Thrasher AJ. Williams DA, et al. Nat Biotechnol. 2011 Jan;29(1):41-2. doi: 10.1038/nbt.1750. Nat Biotechnol. 2011. PMID: 21221100 No abstract available.
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.
Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ. Tolar J, et al. Among authors: williams da. Mol Ther. 2011 Jul;19(7):1193-8. doi: 10.1038/mt.2011.78. Epub 2011 May 3. Mol Ther. 2011. PMID: 21540837 Free PMC article. Review.
Charting a clear path: the ASGCT Standardized Pathways Conference.
Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR. Kiem HP, et al. Among authors: williams da. Mol Ther. 2014 Jul;22(7):1235-1238. doi: 10.1038/mt.2014.95. Mol Ther. 2014. PMID: 24981438 Free PMC article. No abstract available.
Curing genetic disease with gene therapy.
Williams DA. Williams DA. Trans Am Clin Climatol Assoc. 2014;125:122-8; discussion 128-9. Trans Am Clin Climatol Assoc. 2014. PMID: 25125725 Free PMC article.
1,247 results