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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: austin cp. 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: austin cp. 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. Among authors: austin cp. 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.
A call for global action for rare diseases in Africa.
Baynam GS, Groft S, van der Westhuizen FH, Gassman SD, du Plessis K, Coles EP, Selebatso E, Selebatso M, Gaobinelwe B, Selebatso T, Joel D, Llera VA, Vorster BC, Wuebbels B, Djoudalbaye B, Austin CP, Kumuthini J, Forman J, Kaufmann P, Chipeta J, Gavhed D, Larsson A, Stojiljkovic M, Nordgren A, Roldan EJA, Taruscio D, Wong-Rieger D, Nowak K, Bilkey GA, Easteal S, Bowdin S, Reichardt JKV, Beltran S, Kosaki K, van Karnebeek CDM, Gong M, Shuyang Z, Mehrian-Shai R, Adams DR, Puri RD, Zhang F, Pachter N, Muenke M, Nellaker C, Gahl WA, Cederroth H, Broley S, Schoonen M, Boycott KM, Posada M. Baynam GS, et al. Among authors: austin cp. Nat Genet. 2020 Jan;52(1):21-26. doi: 10.1038/s41588-019-0552-2. Nat Genet. 2020. PMID: 31873296 No abstract available.
Improved Diagnosis and Care for Rare Diseases through Implementation of Precision Public Health Framework.
Baynam G, Bowman F, Lister K, Walker CE, Pachter N, Goldblatt J, Boycott KM, Gahl WA, Kosaki K, Adachi T, Ishii K, Mahede T, McKenzie F, Townshend S, Slee J, Kiraly-Borri C, Vasudevan A, Hawkins A, Broley S, Schofield L, Verhoef H, Groza T, Zankl A, Robinson PN, Haendel M, Brudno M, Mattick JS, Dinger ME, Roscioli T, Cowley MJ, Olry A, Hanauer M, Alkuraya FS, Taruscio D, Posada de la Paz M, Lochmüller H, Bushby K, Thompson R, Hedley V, Lasko P, Mina K, Beilby J, Tifft C, Davis M, Laing NG, Julkowska D, Le Cam Y, Terry SF, Kaufmann P, Eerola I, Norstedt I, Rath A, Suematsu M, Groft SC, Austin CP, Draghia-Akli R, Weeramanthri TS, Molster C, Dawkins HJS. Baynam G, et al. Among authors: austin cp. Adv Exp Med Biol. 2017;1031:55-94. doi: 10.1007/978-3-319-67144-4_4. Adv Exp Med Biol. 2017. PMID: 29214566 Review.
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. Among authors: austin cp. 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.
The NCATS Pharmaceutical Collection: a 10-year update.
Huang R, Zhu H, Shinn P, Ngan D, Ye L, Thakur A, Grewal G, Zhao T, Southall N, Hall MD, Simeonov A, Austin CP. Huang R, et al. Among authors: austin cp. Drug Discov Today. 2019 Dec;24(12):2341-2349. doi: 10.1016/j.drudis.2019.09.019. Epub 2019 Oct 1. Drug Discov Today. 2019. PMID: 31585169 Review.
261 results