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Eteplirsen for the treatment of Duchenne muscular dystrophy.
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group. Mendell JR, et al. Among authors: shontz k. Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10. Ann Neurol. 2013. PMID: 23907995 Free article. Clinical Trial.
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: shontz k. 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.
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models.
Sondergaard PC, Griffin DA, Pozsgai ER, Johnson RW, Grose WE, Heller KN, Shontz KM, Montgomery CL, Liu J, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. Sondergaard PC, et al. Ann Clin Transl Neurol. 2015 Mar;2(3):256-70. doi: 10.1002/acn3.172. Epub 2015 Jan 20. Ann Clin Transl Neurol. 2015. PMID: 25815352 Free PMC article.
Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice.
Martin PT, Xu R, Rodino-Klapac LR, Oglesbay E, Camboni M, Montgomery CL, Shontz K, Chicoine LG, Clark KR, Sahenk Z, Mendell JR, Janssen PM. Martin PT, et al. Among authors: shontz k. Am J Physiol Cell Physiol. 2009 Mar;296(3):C476-88. doi: 10.1152/ajpcell.00456.2008. Epub 2008 Dec 24. Am J Physiol Cell Physiol. 2009. PMID: 19109526 Free PMC article.
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: shontz k. 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.
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.
Chicoine LG, Montgomery CL, Bremer WG, Shontz KM, Griffin DA, Heller KN, Lewis S, Malik V, Grose WE, Shilling CJ, Campbell KJ, Preston TJ, Coley BD, Martin PT, Walker CM, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. Chicoine LG, et al. Among authors: shontz km. Mol Ther. 2014 Feb;22(2):338-347. doi: 10.1038/mt.2013.244. Epub 2013 Oct 23. Mol Ther. 2014. PMID: 24196577 Free PMC article.
Safety and Efficacy of AAV Retrograde Pancreatic Ductal Gene Delivery in Normal and Pancreatic Cancer Mice.
Quirin KA, Kwon JJ, Alioufi A, Factora T, Temm CJ, Jacobsen M, Sandusky GE, Shontz K, Chicoine LG, Clark KR, Mendell JT, Korc M, Kota J. Quirin KA, et al. Among authors: shontz k. Mol Ther Methods Clin Dev. 2017 Sep 30;8:8-20. doi: 10.1016/j.omtm.2017.09.006. eCollection 2018 Mar 16. Mol Ther Methods Clin Dev. 2017. PMID: 29349096 Free PMC article.
Partial decellularization eliminates immunogenicity in tracheal allografts.
Tan ZH, Liu L, Dharmadhikari S, Shontz KM, Kreber L, Sperber S, Yu J, Byun WY, Nyirjesy SC, Manning A, Reynolds SD, Chiang T. Tan ZH, et al. Among authors: shontz km. Bioeng Transl Med. 2023 Apr 21;8(5):e10525. doi: 10.1002/btm2.10525. eCollection 2023 Sep. Bioeng Transl Med. 2023. PMID: 37693070 Free PMC article.
32 results