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Fetal gene therapy for neurodegenerative disease of infants.
Massaro G, Mattar CNZ, Wong AMS, Sirka E, Buckley SMK, Herbert BR, Karlsson S, Perocheau DP, Burke D, Heales S, Richard-Londt A, Brandner S, Huebecker M, Priestman DA, Platt FM, Mills K, Biswas A, Cooper JD, Chan JKY, Cheng SH, Waddington SN, Rahim AA. Massaro G, et al. Among authors: priestman da. Nat Med. 2018 Sep;24(9):1317-1323. doi: 10.1038/s41591-018-0106-7. Epub 2018 Jul 16. Nat Med. 2018. PMID: 30013199 Free PMC article.
Imino sugar therapy for type 1 Gaucher disease.
Priestman DA, Platt FM, Dwek RA, Butters TD. Priestman DA, et al. Glycobiology. 2000 Nov;10(11):iv-vi. Glycobiology. 2000. PMID: 11221677 No abstract available.
Infantile-onset symptomatic epilepsy syndrome caused by a homozygous loss-of-function mutation of GM3 synthase.
Simpson MA, Cross H, Proukakis C, Priestman DA, Neville DC, Reinkensmeier G, Wang H, Wiznitzer M, Gurtz K, Verganelaki A, Pryde A, Patton MA, Dwek RA, Butters TD, Platt FM, Crosby AH. Simpson MA, et al. Among authors: priestman da. Nat Genet. 2004 Nov;36(11):1225-9. doi: 10.1038/ng1460. Epub 2004 Oct 24. Nat Genet. 2004. PMID: 15502825 Free article.
Amyotrophic lateral sclerosis and denervation alter sphingolipids and up-regulate glucosylceramide synthase.
Henriques A, Croixmarie V, Priestman DA, Rosenbohm A, Dirrig-Grosch S, D'Ambra E, Huebecker M, Hussain G, Boursier-Neyret C, Echaniz-Laguna A, Ludolph AC, Platt FM, Walther B, Spedding M, Loeffler JP, Gonzalez De Aguilar JL. Henriques A, et al. Among authors: priestman da. Hum Mol Genet. 2015 Dec 20;24(25):7390-405. doi: 10.1093/hmg/ddv439. Epub 2015 Oct 19. Hum Mol Genet. 2015. PMID: 26483191 Free PMC article.
Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.
Kirkegaard T, Gray J, Priestman DA, Wallom KL, Atkins J, Olsen OD, Klein A, Drndarski S, Petersen NH, Ingemann L, Smith DA, Morris L, Bornæs C, Jørgensen SH, Williams I, Hinsby A, Arenz C, Begley D, Jäättelä M, Platt FM. Kirkegaard T, et al. Among authors: priestman da. Sci Transl Med. 2016 Sep 7;8(355):355ra118. doi: 10.1126/scitranslmed.aad9823. Sci Transl Med. 2016. PMID: 27605553 Free PMC article.
62 results