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Surrogate gene therapy for muscular dystrophy.

Davies KE, Chamberlain JS.

Nat Med. 2019 Oct 7. doi: 10.1038/s41591-019-0604-2. [Epub ahead of print] No abstract available.


High levels of AAV vector integration into CRISPR-induced DNA breaks.

Hanlon KS, Kleinstiver BP, Garcia SP, Zaborowski MP, Volak A, Spirig SE, Muller A, Sousa AA, Tsai SQ, Bengtsson NE, Lööv C, Ingelsson M, Chamberlain JS, Corey DP, Aryee MJ, Joung JK, Breakefield XO, Maguire CA, György B.

Nat Commun. 2019 Sep 30;10(1):4439. doi: 10.1038/s41467-019-12449-2.


AAV-based gene therapies for the muscular dystrophies.

Crudele JM, Chamberlain JS.

Hum Mol Genet. 2019 Oct 1;28(R1):R102-R107. doi: 10.1093/hmg/ddz128.


Stabilization of the cardiac sarcolemma by sarcospan rescues DMD-associated cardiomyopathy.

Parvatiyar MS, Brownstein AJ, Kanashiro-Takeuchi RM, Collado JR, Dieseldorff Jones KM, Gopal J, Hammond KG, Marshall JL, Ferrel A, Beedle AM, Chamberlain JS, Renato Pinto J, Crosbie RH.

JCI Insight. 2019 Apr 30;5. pii: 123855. doi: 10.1172/jci.insight.123855.


Development of Novel Micro-dystrophins with Enhanced Functionality.

Ramos JN, Hollinger K, Bengtsson NE, Allen JM, Hauschka SD, Chamberlain JS.

Mol Ther. 2019 Mar 6;27(3):623-635. doi: 10.1016/j.ymthe.2019.01.002. Epub 2019 Feb 1.


Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice.

Kennedy TL, Guiraud S, Edwards B, Squire S, Moir L, Babbs A, Odom G, Golebiowski D, Schneider J, Chamberlain JS, Davies KE.

Mol Ther Methods Clin Dev. 2018 Oct 16;11:92-105. doi: 10.1016/j.omtm.2018.10.005. eCollection 2018 Dec 14.


Non-invasive tracking of disease progression in young dystrophic muscles using multi-parametric MRI at 14T.

Park JS, Vohra R, Klussmann T, Bengtsson NE, Chamberlain JS, Lee D.

PLoS One. 2018 Oct 26;13(10):e0206323. doi: 10.1371/journal.pone.0206323. eCollection 2018.


Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy.

Ho PP, Lahey LJ, Mourkioti F, Kraft PE, Filareto A, Brandt M, Magnusson KEG, Finn EE, Chamberlain JS, Robinson WH, Blau HM, Steinman L.

Proc Natl Acad Sci U S A. 2018 Sep 25;115(39):E9182-E9191. doi: 10.1073/pnas.1808648115. Epub 2018 Sep 4.


Cas9 immunity creates challenges for CRISPR gene editing therapies.

Crudele JM, Chamberlain JS.

Nat Commun. 2018 Aug 29;9(1):3497. doi: 10.1038/s41467-018-05843-9. No abstract available.


Monitoring disease activity noninvasively in the mdx model of Duchenne muscular dystrophy.

Filareto A, Maguire-Nguyen K, Gan Q, Aldanondo G, Machado L, Chamberlain JS, Rando TA.

Proc Natl Acad Sci U S A. 2018 Jul 24;115(30):7741-7746. doi: 10.1073/pnas.1802425115. Epub 2018 Jul 9.


Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins.

Nelson DM, Lindsay A, Judge LM, Duan D, Chamberlain JS, Lowe DA, Ervasti JM.

Hum Mol Genet. 2018 Jun 12. doi: 10.1093/hmg/ddy209. [Epub ahead of print] No abstract available.


Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17.

Adams ME, Odom GL, Kim MJ, Chamberlain JS, Froehner SC.

Hum Mol Genet. 2018 Sep 1;27(17):2978-2985. doi: 10.1093/hmg/ddy197.


Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins.

Nelson DM, Lindsay A, Judge LM, Duan D, Chamberlain JS, Lowe DA, Ervasti JM.

Hum Mol Genet. 2018 Jun 15;27(12):2090-2100. doi: 10.1093/hmg/ddy113. Erratum in: Hum Mol Genet. 2018 Jun 12;:.


AAV6 Vector Production and Purification for Muscle Gene Therapy.

Halbert CL, Allen JM, Chamberlain JS.

Methods Mol Biol. 2018;1687:257-266. doi: 10.1007/978-1-4939-7374-3_18.


A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.

Hakim CH, Wasala NB, Pan X, Kodippili K, Yue Y, Zhang K, Yao G, Haffner B, Duan SX, Ramos J, Schneider JS, Yang NN, Chamberlain JS, Duan D.

Mol Ther Methods Clin Dev. 2017 Jul 27;6:216-230. doi: 10.1016/j.omtm.2017.06.006. eCollection 2017 Sep 15.


Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.

Bengtsson NE, Hall JK, Odom GL, Phelps MP, Andrus CR, Hawkins RD, Hauschka SD, Chamberlain JR, Chamberlain JS.

Nat Commun. 2017 Jun 23;8:16007. doi: 10.1038/ncomms16007.


Translation of Cardiac Myosin Activation with 2-deoxy-ATP to Treat Heart Failure via an Experimental Ribonucleotide Reductase-Based Gene Therapy.

Thomson KS, Odom GL, Murry CE, Mahairas GG, Moussavi-Harami F, Teichman SL, Chen X, Hauschka SD, Chamberlain JS, Regnier M.

JACC Basic Transl Sci. 2016 Dec;1(7):666-679. doi: 10.1016/j.jacbts.2016.07.006.


Progress toward Gene Therapy for Duchenne Muscular Dystrophy.

Chamberlain JR, Chamberlain JS.

Mol Ther. 2017 May 3;25(5):1125-1131. doi: 10.1016/j.ymthe.2017.02.019. Epub 2017 Apr 15. Review.


Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.

Bengtsson NE, Hall JK, Odom GL, Phelps MP, Andrus CR, Hawkins RD, Hauschka SD, Chamberlain JR, Chamberlain JS.

Nat Commun. 2017 Feb 14;8:14454. doi: 10.1038/ncomms14454. Erratum in: Nat Commun. 2017 Jun 23;8:16007.


Loss of niche-satellite cell interactions in syndecan-3 null mice alters muscle progenitor cell homeostasis improving muscle regeneration.

Pisconti A, Banks GB, Babaeijandaghi F, Betta ND, Rossi FM, Chamberlain JS, Olwin BB.

Skelet Muscle. 2016 Oct 4;6:34. eCollection 2016.


Prosurvival Factors Improve Functional Engraftment of Myogenically Converted Dermal Cells into Dystrophic Skeletal Muscle.

Muir LA, Murry CE, Chamberlain JS.

Stem Cells Dev. 2016 Oct;25(20):1559-1569. Epub 2016 Sep 7.


Gene Therapy for Duchenne muscular dystrophy.

Ramos J, Chamberlain JS.

Expert Opin Orphan Drugs. 2015;3(11):1255-1266. Epub 2015 Oct 6.


Progress and prospects of gene therapy clinical trials for the muscular dystrophies.

Bengtsson NE, Seto JT, Hall JK, Chamberlain JS, Odom GL.

Hum Mol Genet. 2016 Apr 15;25(R1):R9-17. doi: 10.1093/hmg/ddv420. Epub 2015 Oct 8. Review.


Viral vector-mediated gene therapies.

Hollinger K, Chamberlain JS.

Curr Opin Neurol. 2015 Oct;28(5):522-7. doi: 10.1097/WCO.0000000000000241. Review.


Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy.

Park J, Wicki J, Knoblaugh SE, Chamberlain JS, Lee D.

PLoS One. 2015 Apr 9;10(4):e0124914. doi: 10.1371/journal.pone.0124914. eCollection 2015.


Recombinant adeno-associated viral (rAAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia.

Su W, Kang J, Sopher B, Gillespie J, Aloi MS, Odom GL, Hopkins S, Case A, Wang DB, Chamberlain JS, Garden GA.

J Neurochem. 2016 Jan;136 Suppl 1:49-62. doi: 10.1111/jnc.13081. Epub 2015 Mar 20.


Perspectives on best practices for gene therapy programs.

Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK.

Hum Gene Ther. 2015 Mar;26(3):127-33. doi: 10.1089/hum.2014.147. Epub 2015 Mar 3.


Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells.

Arnett AL, Konieczny P, Ramos JN, Hall J, Odom G, Yablonka-Reuveni Z, Chamberlain JR, Chamberlain JS.

Mol Ther Methods Clin Dev. 2014;1. pii: 14038.


Engraftment potential of dermal fibroblasts following in vivo myogenic conversion in immunocompetent dystrophic skeletal muscle.

Muir LA, Nguyen QG, Hauschka SD, Chamberlain JS.

Mol Ther Methods Clin Dev. 2014 Jun 25;1:14025.


Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.

Swiderski K, Shaffer SA, Gallis B, Odom GL, Arnett AL, Scott Edgar J, Baum DM, Chee A, Naim T, Gregorevic P, Murphy KT, Moody J, Goodlett DR, Lynch GS, Chamberlain JS.

Hum Mol Genet. 2014 Dec 20;23(25):6697-711. doi: 10.1093/hmg/ddu388. Epub 2014 Jul 31.


Muscle structure influences utrophin expression in mdx mice.

Banks GB, Combs AC, Odom GL, Bloch RJ, Chamberlain JS.

PLoS Genet. 2014 Jun 12;10(6):e1004431. doi: 10.1371/journal.pgen.1004431. eCollection 2014 Jun.


Myofiber branching rather than myofiber hyperplasia contributes to muscle hypertrophy in mdx mice.

Faber RM, Hall JK, Chamberlain JS, Banks GB.

Skelet Muscle. 2014 May 23;4:10. doi: 10.1186/2044-5040-4-10. eCollection 2014.


Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.

Seto JT, Bengtsson NE, Chamberlain JS.

Curr Pediatr Rep. 2014 Jun 1;2(2):102-112.


Mechanical and non-mechanical functions of Dystrophin can prevent cardiac abnormalities in Drosophila.

Taghli-Lamallem O, Jagla K, Chamberlain JS, Bodmer R.

Exp Gerontol. 2014 Jan;49:26-34. doi: 10.1016/j.exger.2013.10.015. Epub 2013 Nov 12.


Removing the immune response from muscular dystrophy research.

Chamberlain JS.

Mol Ther. 2013 Oct;21(10):1821-2. doi: 10.1038/mt.2013.209. No abstract available.


Increased sphingosine-1-phosphate improves muscle regeneration in acutely injured mdx mice.

Ieronimakis N, Pantoja M, Hays AL, Dosey TL, Qi J, Fischer KA, Hoofnagle AN, Sadilek M, Chamberlain JS, Ruohola-Baker H, Reyes M.

Skelet Muscle. 2013 Aug 1;3(1):20. doi: 10.1186/2044-5040-3-20.


Gene and cell-mediated therapies for muscular dystrophy.

Konieczny P, Swiderski K, Chamberlain JS.

Muscle Nerve. 2013 May;47(5):649-63. doi: 10.1002/mus.23738. Epub 2013 Mar 29. Review.


Extracorporeal delivery of rAAV with metabolic exchange and oxygenation.

Bieber S, Halldorson JB, Finn E, Ahmad S, Chamberlain JS, Odom GL.

Sci Rep. 2013;3:1538. doi: 10.1038/srep01538.


An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells.

Filareto A, Parker S, Darabi R, Borges L, Iacovino M, Schaaf T, Mayerhofer T, Chamberlain JS, Ervasti JM, McIvor RS, Kyba M, Perlingeiro RC.

Nat Commun. 2013;4:1549. doi: 10.1038/ncomms2550.


Genetics. A genetic intervention stands a skip away from clinical tests.

Chamberlain JS.

Science. 2012 Dec 14;338(6113):1431-2. doi: 10.1126/science.1233074. No abstract available.


Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction.

Arnett AL, Beutler LR, Quintana A, Allen J, Finn E, Palmiter RD, Chamberlain JS.

Gene Ther. 2013 May;20(5):497-503. doi: 10.1038/gt.2012.60. Epub 2012 Aug 2.


Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin.

Winbanks CE, Weeks KL, Thomson RE, Sepulveda PV, Beyer C, Qian H, Chen JL, Allen JM, Lancaster GI, Febbraio MA, Harrison CA, McMullen JR, Chamberlain JS, Gregorevic P.

J Cell Biol. 2012 Jun 25;197(7):997-1008. doi: 10.1083/jcb.201109091. Epub 2012 Jun 18.


Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.

Wang Z, Storb R, Halbert CL, Banks GB, Butts TM, Finn EE, Allen JM, Miller AD, Chamberlain JS, Tapscott SJ.

Mol Ther. 2012 Aug;20(8):1501-7. doi: 10.1038/mt.2012.111. Epub 2012 Jun 12.


Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors.

Seto JT, Ramos JN, Muir L, Chamberlain JS, Odom GL.

Curr Gene Ther. 2012 Jun;12(3):139-51. Review.


Animal models of muscular dystrophy.

Ng R, Banks GB, Hall JK, Muir LA, Ramos JN, Wicki J, Odom GL, Konieczny P, Seto J, Chamberlain JR, Chamberlain JS.

Prog Mol Biol Transl Sci. 2012;105:83-111. doi: 10.1016/B978-0-12-394596-9.00004-4. Review.


Immune Responses to rAAV6: The Influence of Canine Parvovirus Vaccination and Neonatal Administration of Viral Vector.

Arnett AL, Garikipati D, Wang Z, Tapscott S, Chamberlain JS.

Front Microbiol. 2011 Nov 3;2:220. doi: 10.3389/fmicb.2011.00220. eCollection 2011.


Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials.

Wang Z, Tapscott SJ, Chamberlain JS, Storb R.

Front Microbiol. 2011 Sep 26;2:201. doi: 10.3389/fmicb.2011.00201. eCollection 2011.


Expression of the dystrophin isoform Dp116 preserves functional muscle mass and extends lifespan without preventing dystrophy in severely dystrophic mice.

Judge LM, Arnett AL, Banks GB, Chamberlain JS.

Hum Mol Genet. 2011 Dec 15;20(24):4978-90. doi: 10.1093/hmg/ddr433. Epub 2011 Sep 23.


Age-dependent dystrophin loss and genetic reconstitution establish a molecular link between dystrophin and heart performance during aging.

Townsend D, Daly M, Chamberlain JS, Metzger JM.

Mol Ther. 2011 Oct;19(10):1821-5. doi: 10.1038/mt.2011.120. Epub 2011 Jul 5.


Muscle fiber type-predominant promoter activity in lentiviral-mediated transgenic mouse.

Suga T, Kimura E, Morioka Y, Ikawa M, Li S, Uchino K, Uchida Y, Yamashita S, Maeda Y, Chamberlain JS, Uchino M.

PLoS One. 2011 Mar 18;6(3):e16908. doi: 10.1371/journal.pone.0016908.

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