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Gene therapy for hemophilia.
Nathwani AC. Nathwani AC. Hematology Am Soc Hematol Educ Program. 2019 Dec 6;2019(1):1-8. doi: 10.1182/hematology.2019000007. Hematology Am Soc Hematol Educ Program. 2019. PMID: 31808868 Free PMC article. Review.
Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Chowdary P, et al. N Engl J Med. 2022 Jul 21;387(3):237-247. doi: 10.1056/NEJMoa2119913. N Engl J Med. 2022. PMID: 35857660 Free article. Clinical Trial.
Gene Therapy for Hemophilia.
Nathwani AC, Davidoff AM, Tuddenham EGD. Nathwani AC, et al. Hematol Oncol Clin North Am. 2017 Oct;31(5):853-868. doi: 10.1016/j.hoc.2017.06.011. Hematol Oncol Clin North Am. 2017. PMID: 28895852 Free article. Review.
Gene Therapy for Hemophilia.
Nienhuis AW, Nathwani AC, Davidoff AM. Nienhuis AW, et al. Hum Gene Ther. 2016 Apr;27(4):305-8. doi: 10.1089/hum.2016.018. Hum Gene Ther. 2016. PMID: 27021603 Review.
Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.
Chan JKY, Gil-Farina I, Johana N, Rosales C, Tan YW, Ceiler J, Mcintosh J, Ogden B, Waddington SN, Schmidt M, Biswas A, Choolani M, Nathwani AC, Mattar CNZ. Chan JKY, et al. FASEB J. 2019 Mar;33(3):3954-3967. doi: 10.1096/fj.201801391R. Epub 2018 Dec 5. FASEB J. 2019. PMID: 30517034 Free PMC article.
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA. Paulk NK, et al. Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25. Mol Ther. 2018. PMID: 29055620 Free PMC article.
11 results