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Page 1
Gene therapy for inherited metabolic diseases.
Yilmaz BS, Gurung S, Perocheau D, Counsell J, Baruteau J. Yilmaz BS, et al. J Mother Child. 2020 Nov 10;24(2):53-64. doi: 10.34763/jmotherandchild.20202402si.2004.000009. J Mother Child. 2020. PMID: 33554501 Free PMC article. Review.
Gene therapies targeting the liver.
Cozmescu AC, Counsell J, Gissen P. Cozmescu AC, et al. J Hepatol. 2021 Jan;74(1):235-236. doi: 10.1016/j.jhep.2020.08.003. Epub 2020 Oct 16. J Hepatol. 2021. PMID: 33071009 No abstract available.
Dystrophin involvement in peripheral circadian SRF signalling.
Betts CA, Jagannath A, van Westering TL, Bowerman M, Banerjee S, Meng J, Falzarano MS, Cravo L, McClorey G, Meijboom KE, Bhomra A, Lim WF, Rinaldi C, Counsell JR, Chwalenia K, O'Donovan E, Saleh AF, Gait MJ, Morgan JE, Ferlini A, Foster RG, Wood MJ. Betts CA, et al. Life Sci Alliance. 2021 Aug 13;4(10):e202101014. doi: 10.26508/lsa.202101014. Print 2021 Oct. Life Sci Alliance. 2021. PMID: 34389686 Free PMC article.
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.
An In Vitro Whole-Organ Liver Engineering for Testing of Genetic Therapies.
Lorvellec M, Pellegata AF, Maestri A, Turchetta C, Alvarez Mediavilla E, Shibuya S, Jones B, Scottoni F, Perocheau DP, Cozmescu AC, Delhove JM, Kysh D, Gjinovci A, Counsell JR, Heywood WE, Mills K, McKay TR, De Coppi P, Gissen P. Lorvellec M, et al. iScience. 2020 Nov 13;23(12):101808. doi: 10.1016/j.isci.2020.101808. eCollection 2020 Dec 18. iScience. 2020. PMID: 33305175 Free PMC article.
14 results