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Page 1
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
Tambuyzer E, Vandendriessche B, Austin CP, Brooks PJ, Larsson K, Miller Needleman KI, Valentine J, Davies K, Groft SC, Preti R, Oprea TI, Prunotto M. Tambuyzer E, et al. Among authors: brooks pj. Nat Rev Drug Discov. 2020 Feb;19(2):93-111. doi: 10.1038/s41573-019-0049-9. Epub 2019 Dec 13. Nat Rev Drug Discov. 2020. PMID: 31836861 Review.
Genome editing to 're-write' wrongs.
Perry ME, Valdes KM, Wilder E, Austin CP, Brooks PJ. Perry ME, et al. Among authors: brooks pj. Nat Rev Drug Discov. 2018 Oct;17(10):689-690. doi: 10.1038/nrd.2018.91. Epub 2018 Jun 22. Nat Rev Drug Discov. 2018. PMID: 29930285 No abstract available.
Gene Therapy: The View from NCATS.
Brooks PJ, Yang NN, Austin CP. Brooks PJ, et al. Hum Gene Ther. 2016 Jan;27(1):7-13. doi: 10.1089/hum.2016.29018.pjb. Hum Gene Ther. 2016. PMID: 26784641 Free PMC article. No abstract available.
The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup.
Brooks PJ, Ottinger EA, Portero D, Lomash RM, Alimardanov A, Terse P, Xu X, Chandler RJ, Geist Hauserman J, Esposito E, Bönnemann CG, Venditti CP, Austin CP, Pariser A, Lo DC. Brooks PJ, et al. Hum Gene Ther. 2020 Oct;31(19-20):1034-1042. doi: 10.1089/hum.2020.259. Hum Gene Ther. 2020. PMID: 32993373 Free PMC article. No abstract available.
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.
O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, Montgomery M, Rosenthal E, Samulski RJ, Skarlatos SI, Sorrentino B, Wilson JM, Xie Y, Corrigan-Curay J. O'Reilly M, et al. Among authors: brooks pj. Hum Gene Ther. 2013 Apr;24(4):355-62. doi: 10.1089/hum.2013.064. Hum Gene Ther. 2013. PMID: 23517518 Free PMC article.
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: brooks pj. Nat Rev Drug Discov. 2024 Nov 4. doi: 10.1038/s41573-024-01059-3. Online ahead of print. Nat Rev Drug Discov. 2024. PMID: 39496921 Review.
211 results