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The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.
Jonker AH, Tataru EA, Graessner H, Dimmock D, Jaffe A, Baynam G, Davies J, Mitkus S, Iliach O, Horgan R, Augustine EF, Bateman-House A, Pasmooij AMG, Yu T, Synofzik M, Douville J, Lapteva L, Brooks PJ, O'Connor D, Aartsma-Rus A; N-of-1 Task Force of the International Rare Diseases Research Consortium (IRDiRC). Jonker AH, et al. Among authors: bateman house a. Nat Rev Drug Discov. 2025 Jan;24(1):40-56. doi: 10.1038/s41573-024-01059-3. Epub 2024 Nov 4. Nat Rev Drug Discov. 2025. PMID: 39496921 Review.
Somatic Gene Therapy: Ethics and Access.
Bateman-House A. Bateman-House A. Annu Rev Genomics Hum Genet. 2024 Aug;25(1):421-438. doi: 10.1146/annurev-genom-021623-104458. Annu Rev Genomics Hum Genet. 2024. PMID: 39190912 Review.
Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy.
Cope H, Fischer R, Heslop E, McNiff M, Johnson A, Camino E, Denger B, Armstrong N, Thakrar S, Bateman-House A, Beaverson KL, Woollacott IOC, Phillips D, Fernandez V, Ganot A, Donisa-Dreghici R, Mansfield C, Peay H. Cope H, et al. Among authors: bateman house a. J Neuromuscul Dis. 2024;11(5):1085-1093. doi: 10.3233/JND-240033. J Neuromuscul Dis. 2024. PMID: 39093077 Free PMC article.
ACT To Sustain: Adoptive Cell Therapy To Sustain Access to Non-Commercialized Genetically Modified Cell Therapies.
Gardner RA, White C, Elsallab M, Farnia S, Fraint E, Grilley B, Bateman-House A, Grupp SA, Kenderian S, Locke FL, Nikiforow S, Oluwole OO, Rouce RH, Spiegel J, Shah NN, Sharma A, Komanduri K, Gill S. Gardner RA, et al. Among authors: bateman house a. Transplant Cell Ther. 2024 Aug;30(8):776-787. doi: 10.1016/j.jtct.2024.05.010. Epub 2024 May 16. Transplant Cell Ther. 2024. PMID: 38762057 Review.
The Lived Experience of Pediatric Gene Therapy: A Scoping Review.
Kimberly L, Hunt C, Beaverson K, James E, Bateman-House A, McGowan R, DeSante-Bertkau J. Kimberly L, et al. Among authors: bateman house a. Hum Gene Ther. 2023 Dec;34(23-24):1180-1189. doi: 10.1089/hum.2023.157. Hum Gene Ther. 2023. PMID: 37964764 Review.
50 results