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Page 1
World Federation of Hemophilia Gene Therapy Registry.
Konkle BA, Coffin D, Pierce GF, Clark C, George L, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I; Members of the WFH Gene Therapy Registery Steering Committee. Konkle BA, et al. Among authors: george l. Haemophilia. 2020 Jul;26(4):563-564. doi: 10.1111/hae.14015. Epub 2020 May 27. Haemophilia. 2020. PMID: 32462720 Free PMC article. No abstract available.
Hemophilia gene therapy comes of age.
George LA. George LA. Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):587-594. doi: 10.1182/asheducation-2017.1.587. Hematology Am Soc Hematol Educ Program. 2017. PMID: 29222308 Free PMC article. Review.
Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH.
Konkle B, Pierce G, Coffin D, Naccache M, Clark RC, George L, Iorio A, O'Mahony B, Pipe S, Skinner M, Watson C, Peyvandi F, Mahlangu J; ISTH subcommittee on Factor VIII, Factor IX, rare bleeding disorders. Konkle B, et al. Among authors: george l. J Thromb Haemost. 2020 Nov;18(11):3074-3077. doi: 10.1111/jth.15023. J Thromb Haemost. 2020. PMID: 33463024 Free PMC article.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. George LA, et al. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205. N Engl J Med. 2021. PMID: 34788507 Free PMC article. Clinical Trial.
Hemophilia gene therapy: ushering in a new treatment paradigm?
George LA. George LA. Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):226-233. doi: 10.1182/hematology.2021000254. Hematology Am Soc Hematol Educ Program. 2021. PMID: 34889378 Free PMC article. Review.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.
1,013 results