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Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
Sondhi D, Scott EC, Chen A, Hackett NR, Wong AM, Kubiak A, Nelvagal HR, Pearse Y, Cotman SL, Cooper JD, Crystal RG. Sondhi D, et al. Among authors: nelvagal hr. Hum Gene Ther. 2014 Mar;25(3):223-39. doi: 10.1089/hum.2012.253. Epub 2014 Mar 4. Hum Gene Ther. 2014. PMID: 24372003 Free PMC article.
Towards a new understanding of NCL pathogenesis.
Cooper JD, Tarczyluk MA, Nelvagal HR. Cooper JD, et al. Among authors: nelvagal hr. Biochim Biophys Acta. 2015 Oct;1852(10 Pt B):2256-61. doi: 10.1016/j.bbadis.2015.05.014. Epub 2015 May 28. Biochim Biophys Acta. 2015. PMID: 26026924 Free article. Review.
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: nelvagal hr. 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.
Pathomechanisms in the neuronal ceroid lipofuscinoses.
Nelvagal HR, Lange J, Takahashi K, Tarczyluk-Wells MA, Cooper JD. Nelvagal HR, et al. Biochim Biophys Acta Mol Basis Dis. 2020 Sep 1;1866(9):165570. doi: 10.1016/j.bbadis.2019.165570. Epub 2019 Oct 31. Biochim Biophys Acta Mol Basis Dis. 2020. PMID: 31678162 Free article. Review.
23 results