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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: heasman s. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564. Sci Transl Med. 2021. PMID: 34011628 Free PMC article.
Adult mice are unresponsive to AAV8-Gremlin1 gene therapy targeting the liver.
Khatib Shahidi R, M Hoffmann J, Hedjazifar S, Bonnet L, K Baboota R, Heasman S, Church C, Elias I, Bosch F, Boucher J, Hammarstedt A, Smith U. Khatib Shahidi R, et al. Among authors: heasman s. PLoS One. 2021 Feb 19;16(2):e0247300. doi: 10.1371/journal.pone.0247300. eCollection 2021. PLoS One. 2021. PMID: 33606810 Free PMC article.
Inhibition of T-cell activation by the CTLA4-Fc Abatacept is sufficient to ameliorate proteinuric kidney disease.
Herrera M, Söderberg M, Sabirsh A, Valastro B, Mölne J, Santamaria B, Valverde AM, Guionaud S, Heasman S, Bigley A, Jermutus L, Rondinone C, Coghlan M, Baker D, Quinn CM. Herrera M, et al. Among authors: heasman s. Am J Physiol Renal Physiol. 2017 Apr 1;312(4):F748-F759. doi: 10.1152/ajprenal.00179.2016. Epub 2016 Jul 20. Am J Physiol Renal Physiol. 2017. PMID: 27440778 Free article.
17 results