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Year Number of Results
2019 1
2020 6
2021 8
2022 3
2023 2
2024 0

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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
Adeno-Associated Virus Gene Therapy for Hemophilia.
Samelson-Jones BJ, George LA. Samelson-Jones BJ, et al. Annu Rev Med. 2023 Jan 27;74:231-247. doi: 10.1146/annurev-med-043021-033013. Epub 2022 Sep 14. Annu Rev Med. 2023. PMID: 36103998 Free PMC article. Review.
No CpGs for AAVs?
George LA. George LA. Blood. 2021 Feb 11;137(6):721-723. doi: 10.1182/blood.2020009285. Blood. 2021. PMID: 33570612 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.
Haemophilia care: the only constant is change.
Samelson-Jones BJ, George LA. Samelson-Jones BJ, et al. Br J Haematol. 2021 Sep;194(5):805-807. doi: 10.1111/bjh.17661. Epub 2021 Aug 2. Br J Haematol. 2021. PMID: 34340257 Free PMC article. No abstract available.
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.
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. 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.
Total Knee Arthroplasty after Gene Therapy for Hemophilia B.
Xue F, Wang P, Yuan Z, Shi C, Fang Y, Liu W, Wang Y, Xiao X, Yang R, George LA, Zhang L. Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624. doi: 10.1056/NEJMc2211173. N Engl J Med. 2022. PMID: 36306204 Free PMC article. No abstract available.
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. J Thromb Haemost. 2020 Nov;18(11):3074-3077. doi: 10.1111/jth.15023. J Thromb Haemost. 2020. PMID: 33463024 Free PMC article.
17 results