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In vivo kinetic approach reveals slow SOD1 turnover in the CNS.
Crisp MJ, Mawuenyega KG, Patterson BW, Reddy NC, Chott R, Self WK, Weihl CC, Jockel-Balsarotti J, Varadhachary AS, Bucelli RC, Yarasheski KE, Bateman RJ, Miller TM. Crisp MJ, et al. J Clin Invest. 2015 Jul 1;125(7):2772-80. doi: 10.1172/JCI80705. Epub 2015 Jun 15. J Clin Invest. 2015. PMID: 26075819 Free PMC article.
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.
Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME. Miller TM, et al. Lancet Neurol. 2013 May;12(5):435-42. doi: 10.1016/S1474-4422(13)70061-9. Epub 2013 Mar 29. Lancet Neurol. 2013. PMID: 23541756 Free PMC article. Clinical Trial.