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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: platt fm. 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. Among authors: platt fm. 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: platt fm. Nat Genet. 2004 Nov;36(11):1225-9. doi: 10.1038/ng1460. Epub 2004 Oct 24. Nat Genet. 2004. PMID: 15502825 Free article.
Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease.
Lee JP, Jeyakumar M, Gonzalez R, Takahashi H, Lee PJ, Baek RC, Clark D, Rose H, Fu G, Clarke J, McKercher S, Meerloo J, Muller FJ, Park KI, Butters TD, Dwek RA, Schwartz P, Tong G, Wenger D, Lipton SA, Seyfried TN, Platt FM, Snyder EY. Lee JP, et al. Among authors: platt fm. Nat Med. 2007 Apr;13(4):439-47. doi: 10.1038/nm1548. Epub 2007 Mar 11. Nat Med. 2007. PMID: 17351625
β-Glucosidase 2 (GBA2) activity and imino sugar pharmacology.
Ridley CM, Thur KE, Shanahan J, Thillaiappan NB, Shen A, Uhl K, Walden CM, Rahim AA, Waddington SN, Platt FM, van der Spoel AC. Ridley CM, et al. Among authors: platt fm. J Biol Chem. 2013 Sep 6;288(36):26052-26066. doi: 10.1074/jbc.M113.463562. Epub 2013 Jul 23. J Biol Chem. 2013. PMID: 23880767 Free PMC article.
257 results