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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. Among authors: privolizzi r. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564. Sci Transl Med. 2021. PMID: 34011628 Free PMC article.
A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells.
Waddington SN, Privolizzi R, Karda R, O'Neill HC. Waddington SN, et al. Among authors: privolizzi r. Curr Stem Cell Rep. 2016;2(1):9-20. doi: 10.1007/s40778-016-0037-5. Epub 2016 Feb 11. Curr Stem Cell Rep. 2016. PMID: 27398291 Free PMC article. Review.