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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: dearborn jt. J Clin Invest. 2022 Oct 17;132(20):e163107. doi: 10.1172/JCI163107. J Clin Invest. 2022. PMID: 36040802 Free PMC article.
An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis.
Macauley SL, Wong AM, Shyng C, Augner DP, Dearborn JT, Pearse Y, Roberts MS, Fowler SC, Cooper JD, Watterson DM, Sands MS. Macauley SL, et al. Among authors: dearborn jt. J Neurosci. 2014 Sep 24;34(39):13077-82. doi: 10.1523/JNEUROSCI.2518-14.2014. J Neurosci. 2014. PMID: 25253854 Free PMC article.
Synergistic effects of treating the spinal cord and brain in CLN1 disease.
Shyng C, Nelvagal HR, Dearborn JT, Tyynelä J, Schmidt RE, Sands MS, Cooper JD. Shyng C, et al. Among authors: dearborn jt. Proc Natl Acad Sci U S A. 2017 Jul 18;114(29):E5920-E5929. doi: 10.1073/pnas.1701832114. Epub 2017 Jul 3. Proc Natl Acad Sci U S A. 2017. PMID: 28673981 Free PMC article.
Cell-autonomous expression of the acid hydrolase galactocerebrosidase.
Mikulka CR, Dearborn JT, Benitez BA, Strickland A, Liu L, Milbrandt J, Sands MS. Mikulka CR, et al. Among authors: dearborn jt. Proc Natl Acad Sci U S A. 2020 Apr 21;117(16):9032-9041. doi: 10.1073/pnas.1917675117. Epub 2020 Apr 6. Proc Natl Acad Sci U S A. 2020. PMID: 32253319 Free PMC article.
31 results