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Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep.
Nelvagal HR, Eaton SL, Wang SH, Eultgen EM, Takahashi K, Le SQ, Nesbitt R, Dearborn JT, Siano N, Puhl AC, Dickson PI, Thompson G, Murdoch F, Brennan PM, Gray M, Greenhalgh SN, Tennant P, Gregson R, Clutton E, Nixon J, Proudfoot C, Guido S, Lillico SG, Whitelaw CBA, Lu JY, Hofmann SL, Ekins S, Sands MS, Wishart TM, Cooper JD. Nelvagal HR, et al. Among authors: wang sh. J Clin Invest. 2022 Oct 17;132(20):e163107. doi: 10.1172/JCI163107. J Clin Invest. 2022. PMID: 36040802 Free PMC article.
Severe central nervous system demyelination in Sanfilippo disease.
Taherzadeh M, Zhang E, Londono I, De Leener B, Wang S, Cooper JD, Kennedy TE, Morales CR, Chen Z, Lodygensky GA, Pshezhetsky AV. Taherzadeh M, et al. Front Mol Neurosci. 2023 Dec 13;16:1323449. doi: 10.3389/fnmol.2023.1323449. eCollection 2023. Front Mol Neurosci. 2023. PMID: 38163061 Free PMC article.
GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice.
Takahashi K, Rensing NR, Eultgen EM, Wang SH, Nelvagal HR, Le SQ, Roberts MS, Doray B, Han EB, Dickson PI, Wong M, Sands MS, Cooper JD. Takahashi K, et al. Among authors: wang sh. bioRxiv [Preprint]. 2024 Mar 30:2024.03.29.587276. doi: 10.1101/2024.03.29.587276. bioRxiv. 2024. PMID: 38585903 Free PMC article. Preprint.
2,609 results