Rodino-Klapac LR - Search Results

1

Current Clinical Applications of In Vivo Gene Therapy with AAVs.

Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.

2

Gene therapy for duchenne muscular dystrophy: expectations and challenges.

Rodino-Klapac LR, Chicoine LG, Kaspar BK, Mendell JR. Rodino-Klapac LR, et al. Arch Neurol. 2007 Sep;64(9):1236-41. doi: 10.1001/archneur.64.9.1236. Arch Neurol. 2007. PMID: 17846262 Review.

3

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy.

Rodino-Klapac LR, Janssen PM, Montgomery CL, Coley BD, Chicoine LG, Clark KR, Mendell JR. Rodino-Klapac LR, et al. J Transl Med. 2007 Sep 24;5:45. doi: 10.1186/1479-5876-5-45. J Transl Med. 2007. PMID: 17892583 Free PMC article.

4

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR. Rodino-Klapac LR, et al. Neurology. 2008 Jul 22;71(4):240-7. doi: 10.1212/01.wnl.0000306309.85301.e2. Epub 2008 Jun 4. Neurology. 2008. PMID: 18525034

5

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. 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.

6

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease.

Rodino-Klapac LR, Haidet AM, Kota J, Handy C, Kaspar BK, Mendell JR. Rodino-Klapac LR, et al. Muscle Nerve. 2009 Mar;39(3):283-96. doi: 10.1002/mus.21244. Muscle Nerve. 2009. PMID: 19208403 Free PMC article. Review.

7

Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.

Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR. Mendell JR, et al. Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732. Ann Neurol. 2009. PMID: 19798725 Free PMC article. Clinical Trial.

8

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery.

Rodino-Klapac LR, Montgomery CL, Bremer WG, Shontz KM, Malik V, Davis N, Sprinkle S, Campbell KJ, Sahenk Z, Clark KR, Walker CM, Mendell JR, Chicoine LG. Rodino-Klapac LR, et al. Mol Ther. 2010 Jan;18(1):109-17. doi: 10.1038/mt.2009.254. Epub 2009 Nov 10. Mol Ther. 2010. PMID: 19904237 Free PMC article.

9

Follistatin gene delivery enhances muscle growth and strength in nonhuman primates.

Kota J, Handy CR, Haidet AM, Montgomery CL, Eagle A, Rodino-Klapac LR, Tucker D, Shilling CJ, Therlfall WR, Walker CM, Weisbrode SE, Janssen PM, Clark KR, Sahenk Z, Mendell JR, Kaspar BK. Kota J, et al. Sci Transl Med. 2009 Nov 11;1(6):6ra15. doi: 10.1126/scitranslmed.3000112. Sci Transl Med. 2009. PMID: 20368179 Free PMC article.

10

Molecular therapeutic strategies targeting Duchenne muscular dystrophy.

Mendell JR, Rodino-Klapac LR, Malik V. Mendell JR, et al. J Child Neurol. 2010 Sep;25(9):1145-8. doi: 10.1177/0883073810371005. Epub 2010 May 24. J Child Neurol. 2010. PMID: 20498331 Free PMC article. Review.

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