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Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy.
Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM. Fu H, et al. Among authors: flanigan km. Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24. Hum Gene Ther Clin Dev. 2017. PMID: 29064732 Free PMC article.
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.
Murrey DA, Naughton BJ, Duncan FJ, Meadows AS, Ware TA, Campbell KJ, Bremer WG, Walker CM, Goodchild L, Bolon B, La Perle K, Flanigan KM, McBride KL, McCarty DM, Fu H. Murrey DA, et al. Among authors: flanigan km. Hum Gene Ther Clin Dev. 2014 Jun;25(2):72-84. doi: 10.1089/humc.2013.208. Epub 2014 Apr 10. Hum Gene Ther Clin Dev. 2014. PMID: 24720466 Free PMC article.
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, Al-Zaidy S, Govoni A, Rodino-Klapac LR, Hogan MJ, Kaspar BK. Mendell JR, et al. Among authors: flanigan km. Mol Ther. 2015 Jan;23(1):192-201. doi: 10.1038/mt.2014.200. Epub 2014 Oct 17. Mol Ther. 2015. PMID: 25322757 Free PMC article. Clinical Trial.
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.
Meadows AS, Duncan FJ, Camboni M, Waligura K, Montgomery C, Zaraspe K, Naughton BJ, Bremer WG, Shilling C, Walker CM, Bolon B, Flanigan KM, McBride KL, McCarty DM, Fu H. Meadows AS, et al. Among authors: flanigan km. Hum Gene Ther Clin Dev. 2015 Dec;26(4):228-42. doi: 10.1089/humc.2015.132. Hum Gene Ther Clin Dev. 2015. PMID: 26684447 Free PMC article.
A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design.
Truxal KV, Fu H, McCarty DM, McNally KA, Kunkler KL, Zumberge NA, Martin L, Aylward SC, Alfano LN, Berry KM, Lowes LP, Corridore M, McKee C, McBride KL, Flanigan KM. Truxal KV, et al. Among authors: flanigan km. Mol Genet Metab. 2016 Nov;119(3):239-248. doi: 10.1016/j.ymgme.2016.08.002. Epub 2016 Aug 18. Mol Genet Metab. 2016. PMID: 27590925
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, Lewis S, McColly M, Hogan MJ, Kaspar BK. Mendell JR, et al. Among authors: flanigan km. Mol Ther. 2017 Apr 5;25(4):870-879. doi: 10.1016/j.ymthe.2017.02.015. Epub 2017 Mar 6. Mol Ther. 2017. PMID: 28279643 Free PMC article.
189 results