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Cited In for PMID: 24002329

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Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.
Belur LR, Temme A, Podetz-Pedersen KM, Riedl M, Vulchanova L, Robinson N, Hanson LR, Kozarsky KF, Orchard PJ, Frey WH 2nd, Low WC, McIvor RS. Belur LR, et al. Hum Gene Ther. 2017 Jul;28(7):576-587. doi: 10.1089/hum.2017.187. Epub 2017 Apr 20. Hum Gene Ther. 2017. PMID: 28462595 Free PMC article.
Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.
Dickson PI, Kaitila I, Harmatz P, Mlikotic A, Chen AH, Victoroff A, Passage MB, Madden J, Le SQ, Naylor DE; Mucopolysaccharidosis I Intrathecal Research Collaborative. Dickson PI, et al. Mol Genet Metab. 2015 Sep-Oct;116(1-2):69-74. doi: 10.1016/j.ymgme.2015.07.005. Epub 2015 Jul 26. Mol Genet Metab. 2015. PMID: 26260077 Free PMC article. Clinical Trial.
13 results