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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: burke d. 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.
Proteomic Discovery and Development of a Multiplexed Targeted MRM-LC-MS/MS Assay for Urine Biomarkers of Extracellular Matrix Disruption in Mucopolysaccharidoses I, II, and VI.
Heywood WE, Camuzeaux S, Doykov I, Patel N, Preece RL, Footitt E, Cleary M, Clayton P, Grunewald S, Abulhoul L, Chakrapani A, Sebire NJ, Hindmarsh P, de Koning TJ, Heales S, Burke D, Gissen P, Mills K. Heywood WE, et al. Among authors: burke d. Anal Chem. 2015 Dec 15;87(24):12238-44. doi: 10.1021/acs.analchem.5b03232. Epub 2015 Nov 20. Anal Chem. 2015. PMID: 26537538
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.
Baruteau J, Cunningham SC, Yilmaz BS, Perocheau DP, Eaglestone S, Burke D, Thrasher AJ, Waddington SN, Lisowski L, Alexander IE, Gissen P. Baruteau J, et al. Among authors: burke d. Mol Ther Methods Clin Dev. 2021 Sep 14;23:135-146. doi: 10.1016/j.omtm.2021.09.005. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34703837 Free PMC article.
Cerebellar and Midbrain Lysosomal Enzyme Deficiency in Isolated Dystonia.
Schreglmann SR, Burke D, Batla A, Kresojevic N, Wood N, Heales S, Bhatia KP. Schreglmann SR, et al. Among authors: burke d. Mov Disord. 2022 Apr;37(4):875-877. doi: 10.1002/mds.28937. Epub 2022 Jan 25. Mov Disord. 2022. PMID: 35080042 No abstract available.
2,337 results