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Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.
Ng J, Barral S, De La Fuente Barrigon C, Lignani G, Erdem FA, Wallings R, Privolizzi R, Rossignoli G, Alrashidi H, Heasman S, Meyer E, Ngoh A, Pope S, Karda R, Perocheau D, Baruteau J, Suff N, Antinao Diaz J, Schorge S, Vowles J, Marshall LR, Cowley SA, Sucic S, Freissmuth M, Counsell JR, Wade-Martins R, Heales SJR, Rahim AA, Bencze M, Waddington SN, Kurian MA. Ng J, et al. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564. Sci Transl Med. 2021. PMID: 34011628 Free PMC article.
Episomal transgene expression in pluripotent stem cells.
Lufino MM, Popplestone AR, Cowley SA, Edser PA, James WS, Wade-Martins R. Lufino MM, et al. Methods Mol Biol. 2011;767:369-87. doi: 10.1007/978-1-61779-201-4_27. Methods Mol Biol. 2011. PMID: 21822889
Physiological characterisation of human iPS-derived dopaminergic neurons.
Hartfield EM, Yamasaki-Mann M, Ribeiro Fernandes HJ, Vowles J, James WS, Cowley SA, Wade-Martins R. Hartfield EM, et al. PLoS One. 2014 Feb 21;9(2):e87388. doi: 10.1371/journal.pone.0087388. eCollection 2014. PLoS One. 2014. PMID: 24586273 Free PMC article.
LRRK2 BAC transgenic rats develop progressive, L-DOPA-responsive motor impairment, and deficits in dopamine circuit function.
Sloan M, Alegre-Abarrategui J, Potgieter D, Kaufmann AK, Exley R, Deltheil T, Threlfell S, Connor-Robson N, Brimblecombe K, Wallings R, Cioroch M, Bannerman DM, Bolam JP, Magill PJ, Cragg SJ, Dodson PD, Wade-Martins R. Sloan M, et al. Hum Mol Genet. 2016 Mar 1;25(5):951-63. doi: 10.1093/hmg/ddv628. Epub 2016 Jan 6. Hum Mol Genet. 2016. PMID: 26744332 Free PMC article.
ER Stress and Autophagic Perturbations Lead to Elevated Extracellular α-Synuclein in GBA-N370S Parkinson's iPSC-Derived Dopamine Neurons.
Fernandes HJ, Hartfield EM, Christian HC, Emmanoulidou E, Zheng Y, Booth H, Bogetofte H, Lang C, Ryan BJ, Sardi SP, Badger J, Vowles J, Evetts S, Tofaris GK, Vekrellis K, Talbot K, Hu MT, James W, Cowley SA, Wade-Martins R. Fernandes HJ, et al. Stem Cell Reports. 2016 Mar 8;6(3):342-56. doi: 10.1016/j.stemcr.2016.01.013. Epub 2016 Feb 18. Stem Cell Reports. 2016. PMID: 26905200 Free PMC article.
C9orf72 Hexanucleotide Expansions Are Associated with Altered Endoplasmic Reticulum Calcium Homeostasis and Stress Granule Formation in Induced Pluripotent Stem Cell-Derived Neurons from Patients with Amyotrophic Lateral Sclerosis and Frontotemporal Dementia.
Dafinca R, Scaber J, Ababneh N, Lalic T, Weir G, Christian H, Vowles J, Douglas AG, Fletcher-Jones A, Browne C, Nakanishi M, Turner MR, Wade-Martins R, Cowley SA, Talbot K. Dafinca R, et al. Stem Cells. 2016 Aug;34(8):2063-78. doi: 10.1002/stem.2388. Epub 2016 May 4. Stem Cells. 2016. PMID: 27097283 Free PMC article.
170 results