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Absence of full-length dystrophin impairs normal maturation and contraction of cardiomyocytes derived from human induced pluripotent stem cells.

Pioner JM, Guan X, Klaiman JM, Racca AW, Pabon L, Muskheli V, Macadangdang J, Ferrantini C, Hoopmann MR, Moritz RL, Kim DH, Tesi C, Poggesi C, Murry CE, Childers MK, Mack DL, Regnier M.

Cardiovasc Res. 2019 May 2. pii: cvz109. doi: 10.1093/cvr/cvz109. [Epub ahead of print]


Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy.

Aminzadeh MA, Rogers RG, Fournier M, Tobin RE, Guan X, Childers MK, Andres AM, Taylor DJ, Ibrahim A, Ding X, Torrente A, Goldhaber JM, Lewis M, Gottlieb RA, Victor RA, Marbán E.

Stem Cell Reports. 2018 Mar 13;10(3):942-955. doi: 10.1016/j.stemcr.2018.01.023. Epub 2018 Mar 1.


Isolation and characterization of canine perivascular stem/stromal cells for bone tissue engineering.

James AW, Zhang X, Crisan M, Hardy WR, Liang P, Meyers CA, Lobo S, Lagishetty V, Childers MK, Asatrian G, Ding C, Yen YH, Zou E, Ting K, Peault B, Soo C.

PLoS One. 2017 May 10;12(5):e0177308. doi: 10.1371/journal.pone.0177308. eCollection 2017.


Cardiac involvement in female carriers of Duchenne or Becker muscular dystrophy.

Childers MK, Klaiman JM.

Muscle Nerve. 2017 Jun;55(6):777-779. doi: 10.1002/mus.25661. Epub 2017 May 5. No abstract available.


Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.

Elverman M, Goddard MA, Mack D, Snyder JM, Lawlor MW, Meng H, Beggs AH, Buj-Bello A, Poulard K, Marsh AP, Grange RW, Kelly VE, Childers MK.

Muscle Nerve. 2017 Nov;56(5):943-953. doi: 10.1002/mus.25658. Epub 2017 May 22.


Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.

Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, Grange RW, Elverman MR, Denard J, Veron P, Buscara L, Le Bec C, Hogrel JY, Brezovec AG, Meng H, Yang L, Liu F, O'Callaghan M, Gopal N, Kelly VE, Smith BK, Strande JL, Mavilio F, Beggs AH, Mingozzi F, Lawlor MW, Buj-Bello A, Childers MK.

Mol Ther. 2017 Apr 5;25(4):839-854. doi: 10.1016/j.ymthe.2017.02.004. Epub 2017 Feb 22.


The utility of genetic testing in neuromuscular disease: A consensus statement from the AANEM on the clinical utility of genetic testing in diagnosis of neuromuscular disease.

Kassardjian CD, Amato AA, Boon AJ, Childers MK, Klein CJ; AANEM Professional Practice Committee.

Muscle Nerve. 2016 Dec;54(6):1007-1009. doi: 10.1002/mus.25387.


Isolation and Mechanical Measurements of Myofibrils from Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes.

Pioner JM, Racca AW, Klaiman JM, Yang KC, Guan X, Pabon L, Muskheli V, Zaunbrecher R, Macadangdang J, Jeong MY, Mack DL, Childers MK, Kim DH, Tesi C, Poggesi C, Murry CE, Regnier M.

Stem Cell Reports. 2016 Jun 14;6(6):885-896. doi: 10.1016/j.stemcr.2016.04.006. Epub 2016 May 5.


Nicorandil, a Nitric Oxide Donor and ATP-Sensitive Potassium Channel Opener, Protects Against Dystrophin-Deficient Cardiomyopathy.

Afzal MZ, Reiter M, Gastonguay C, McGivern JV, Guan X, Ge ZD, Mack DL, Childers MK, Ebert AD, Strande JL.

J Cardiovasc Pharmacol Ther. 2016 Nov;21(6):549-562. Epub 2016 Mar 2.


Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons.

Lawlor MW, Beggs AH, Buj-Bello A, Childers MK, Dowling JJ, James ES, Meng H, Moore SA, Prasad S, Schoser B, Sewry CA.

J Neuropathol Exp Neurol. 2016 Feb;75(2):102-10. Review. Erratum in: J Neuropathol Exp Neurol. 2016 May;75(5):479.


Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myopathy.

Goddard MA, Mack DL, Czerniecki SM, Kelly VE, Snyder JM, Grange RW, Lawlor MW, Smith BK, Beggs AH, Childers MK.

Ann Transl Med. 2015 Oct;3(18):262. doi: 10.3978/j.issn.2305-5839.2015.10.31. Erratum in: Ann Transl Med. 2016 Jan;4(1):23.


Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs.

Childers MK, Beggs AH, Buj-Bello A.

Ann Transl Med. 2015 Oct;3(17):257. doi: 10.3978/j.issn.2305-5839.2015.10.01. No abstract available.


Gene therapy in monogenic congenital myopathies.

Guan X, Goddard MA, Mack DL, Childers MK.

Methods. 2016 Apr 15;99:91-8. doi: 10.1016/j.ymeth.2015.10.004. Epub 2015 Oct 14. Review.


Nanopatterned Human iPSC-based Model of a Dystrophin-Null Cardiomyopathic Phenotype.

Macadangdang J, Guan X, Smith AS, Lucero R, Czerniecki S, Childers MK, Mack DL, Kim DH.

Cell Mol Bioeng. 2015 Sep;8(3):320-332.


Use of Adeno-Associated Virus to Enrich Cardiomyocytes Derived from Human Stem Cells.

Guan X, Wang Z, Czerniecki S, Mack D, François V, Blouin V, Moullier P, Childers MK.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):194-201. doi: 10.1089/humc.2015.052. Epub 2015 Aug 7.


Validity of a Neurological Scoring System for Canine X-Linked Myotubular Myopathy.

Snyder JM, Meisner A, Mack D, Goddard M, Coulter IT, Grange R, Childers MK.

Hum Gene Ther Clin Dev. 2015 Jun;26(2):131-7. doi: 10.1089/humc.2015.049.


Guiding intramuscular diaphragm injections using real-time ultrasound and electromyography.

Sarwal A, Cartwright MS, Mitchell E, Williams K, Walker FO, Childers MK.

Muscle Nerve. 2015 Feb;51(2):287-9. doi: 10.1002/mus.24493.


Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.

Braun R, Wang Z, Mack DL, Childers MK.

Am J Phys Med Rehabil. 2014 Nov;93(11 Suppl 3):S97-107. doi: 10.1097/PHM.0000000000000138. Review.


Regenerative rehabilitation: a new future?

Perez-Terzic C, Childers MK.

Am J Phys Med Rehabil. 2014 Nov;93(11 Suppl 3):S73-8. doi: 10.1097/PHM.0000000000000211.


Gait characteristics in a canine model of X-linked myotubular myopathy.

Goddard MA, Burlingame E, Beggs AH, Buj-Bello A, Childers MK, Marsh AP, Kelly VE.

J Neurol Sci. 2014 Nov 15;346(1-2):221-6. doi: 10.1016/j.jns.2014.08.032. Epub 2014 Aug 29.


Syngeneic Myoblast Transplantation Improves Muscle Function in a Murine Model of X-Linked Myotubular Myopathy.

Lim HJ, Joo S, Oh SH, Jackson JD, Eckman DM, Bledsoe TM, Pierson CR, Childers MK, Atala A, Yoo JJ.

Cell Transplant. 2015;24(9):1887-900. doi: 10.3727/096368914X683494. Epub 2014 Jul 25.


Disease-in-a-dish: the contribution of patient-specific induced pluripotent stem cell technology to regenerative rehabilitation.

Mack DL, Guan X, Wagoner A, Walker SJ, Childers MK.

Am J Phys Med Rehabil. 2014 Nov;93(11 Suppl 3):S155-68. doi: 10.1097/PHM.0000000000000141. Review.


Tissue triage and freezing for models of skeletal muscle disease.

Meng H, Janssen PM, Grange RW, Yang L, Beggs AH, Swanson LC, Cossette SA, Frase A, Childers MK, Granzier H, Gussoni E, Lawlor MW.

J Vis Exp. 2014 Jul 15;(89). doi: 10.3791/51586.


Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy.

Sarwal A, Cartwright MS, Walker FO, Mitchell E, Buj-Bello A, Beggs AH, Childers MK.

Muscle Nerve. 2014 Oct;50(4):607-9. doi: 10.1002/mus.24294. Epub 2014 Aug 30.


Respiratory assessment in centronuclear myopathies.

Smith BK, Goddard M, Childers MK.

Muscle Nerve. 2014 Sep;50(3):315-26. doi: 10.1002/mus.24249. Epub 2014 Aug 5. Review.


Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Childers MK, Joubert R, Poulard K, Moal C, Grange RW, Doering JA, Lawlor MW, Rider BE, Jamet T, Danièle N, Martin S, Rivière C, Soker T, Hammer C, Van Wittenberghe L, Lockard M, Guan X, Goddard M, Mitchell E, Barber J, Williams JK, Mack DL, Furth ME, Vignaud A, Masurier C, Mavilio F, Moullier P, Beggs AH, Buj-Bello A.

Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523.


Dystrophin-deficient cardiomyocytes derived from human urine: new biologic reagents for drug discovery.

Guan X, Mack DL, Moreno CM, Strande JL, Mathieu J, Shi Y, Markert CD, Wang Z, Liu G, Lawlor MW, Moorefield EC, Jones TN, Fugate JA, Furth ME, Murry CE, Ruohola-Baker H, Zhang Y, Santana LF, Childers MK.

Stem Cell Res. 2014 Mar;12(2):467-80. doi: 10.1016/j.scr.2013.12.004. Epub 2013 Dec 23.


Diaphragm remodeling and compensatory respiratory mechanics in a canine model of Duchenne muscular dystrophy.

Mead AF, Petrov M, Malik AS, Mitchell MA, Childers MK, Bogan JR, Seidner G, Kornegay JN, Stedman HH.

J Appl Physiol (1985). 2014 Apr 1;116(7):807-15. doi: 10.1152/japplphysiol.00833.2013. Epub 2014 Jan 9.


Selenoprotein N deficiency in mice is associated with abnormal lung development.

Moghadaszadeh B, Rider BE, Lawlor MW, Childers MK, Grange RW, Gupta K, Boukedes SS, Owen CA, Beggs AH.

FASEB J. 2013 Apr;27(4):1585-99. doi: 10.1096/fj.12-212688. Epub 2013 Jan 16.


Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy.

Lawlor MW, Armstrong D, Viola MG, Widrick JJ, Meng H, Grange RW, Childers MK, Hsu CP, O'Callaghan M, Pierson CR, Buj-Bello A, Beggs AH.

Hum Mol Genet. 2013 Apr 15;22(8):1525-38. doi: 10.1093/hmg/ddt003. Epub 2013 Jan 9.


Muscle function in a canine model of X-linked myotubular myopathy.

Grange RW, Doering J, Mitchell E, Holder MN, Guan X, Goddard M, Tegeler C, Beggs AH, Childers MK.

Muscle Nerve. 2012 Oct;46(4):588-91. doi: 10.1002/mus.23463.


Increasing need for academic leadership in clinical trials.

Childers MK.

PM R. 2012 Jun;4(6):391-3. doi: 10.1016/j.pmrj.2012.04.002. No abstract available.


Chronic administration of a leupeptin-derived calpain inhibitor fails to ameliorate severe muscle pathology in a canine model of duchenne muscular dystrophy.

Childers MK, Bogan JR, Bogan DJ, Greiner H, Holder M, Grange RW, Kornegay JN.

Front Pharmacol. 2012 Jan 9;2:89. doi: 10.3389/fphar.2011.00089. eCollection 2011.


The paradox of muscle hypertrophy in muscular dystrophy.

Kornegay JN, Childers MK, Bogan DJ, Bogan JR, Nghiem P, Wang J, Fan Z, Howard JF Jr, Schatzberg SJ, Dow JL, Grange RW, Styner MA, Hoffman EP, Wagner KR.

Phys Med Rehabil Clin N Am. 2012 Feb;23(1):149-72, xii. doi: 10.1016/j.pmr.2011.11.014. Review.


Establishing clinical end points of respiratory function in large animals for clinical translation.

Goddard MA, Mitchell EL, Smith BK, Childers MK.

Phys Med Rehabil Clin N Am. 2012 Feb;23(1):75-94, xi. doi: 10.1016/j.pmr.2011.11.017. Epub 2011 Dec 15.


Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies.

Kornegay JN, Bogan JR, Bogan DJ, Childers MK, Li J, Nghiem P, Detwiler DA, Larsen CA, Grange RW, Bhavaraju-Sanka RK, Tou S, Keene BP, Howard JF Jr, Wang J, Fan Z, Schatzberg SJ, Styner MA, Flanigan KM, Xiao X, Hoffman EP.

Mamm Genome. 2012 Feb;23(1-2):85-108. doi: 10.1007/s00335-011-9382-y. Epub 2012 Jan 5. Review.


Aging and physical mobility in group-housed Old World monkeys.

Shively CA, Willard SL, Register TC, Bennett AJ, Pierre PJ, Laudenslager ML, Kitzman DW, Childers MK, Grange RW, Kritchevsky SB.

Age (Dordr). 2012 Oct;34(5):1123-31. Epub 2011 Dec 28.


Stem cell use in musculoskeletal disorders.

Guan X, Furth ME, Childers MK.

PM R. 2011 Jun;3(6 Suppl 1):S95-9. doi: 10.1016/j.pmrj.2011.04.002. Review.


In vivo canine muscle function assay.

Childers MK, Grange RW, Kornegay JN.

J Vis Exp. 2011 Apr 5;(50). pii: 2623. doi: 10.3791/2623.


Golden retriever muscular dystrophy (GRMD): Developing and maintaining a colony and physiological functional measurements.

Kornegay JN, Bogan JR, Bogan DJ, Childers MK, Grange RW.

Methods Mol Biol. 2011;709:105-23. doi: 10.1007/978-1-61737-982-6_7.


MTM1 mutation associated with X-linked myotubular myopathy in Labrador Retrievers.

Beggs AH, Böhm J, Snead E, Kozlowski M, Maurer M, Minor K, Childers MK, Taylor SM, Hitte C, Mickelson JR, Guo LT, Mizisin AP, Buj-Bello A, Tiret L, Laporte J, Shelton GD.

Proc Natl Acad Sci U S A. 2010 Aug 17;107(33):14697-702. doi: 10.1073/pnas.1003677107. Epub 2010 Aug 3.


A single-dose resveratrol treatment in a mouse model of amyotrophic lateral sclerosis.

Markert CD, Kim E, Gifondorwa DJ, Childers MK, Milligan CE.

J Med Food. 2010 Oct;13(5):1081-5. doi: 10.1089/jmf.2009.0243.


Aquatic versus land-based exercises as early functional rehabilitation for elite athletes with acute lower extremity ligament injury: a pilot study.

Kim E, Kim T, Kang H, Lee J, Childers MK.

PM R. 2010 Aug;2(8):703-12. doi: 10.1016/j.pmrj.2010.03.012. Epub 2010 Jul 3.


Eccentric contractions induce rapid isometric torque drop in dystrophin-deficient dogs.

Tegeler CJ, Grange RW, Bogan DJ, Markert CD, Case D, Kornegay JN, Childers MK.

Muscle Nerve. 2010 Jul;42(1):130-2. doi: 10.1002/mus.21699.


Functional muscle analysis of the Tcap knockout mouse.

Markert CD, Meaney MP, Voelker KA, Grange RW, Dalley HW, Cann JK, Ahmed M, Bishwokarma B, Walker SJ, Yu SX, Brown M, Lawlor MW, Beggs AH, Childers MK.

Hum Mol Genet. 2010 Jun 1;19(11):2268-83. doi: 10.1093/hmg/ddq105. Epub 2010 Mar 16.


Kinematics of gait in golden retriever muscular dystrophy.

Marsh AP, Eggebeen JD, Kornegay JN, Markert CD, Childers MK.

Neuromuscul Disord. 2010 Jan;20(1):16-20. doi: 10.1016/j.nmd.2009.10.007. Epub 2009 Nov 22.


Immunofluorescence microscopy for imaging of nuclear p63 in human primary keratinocytes: a comparison of antibodies and fixation methods.

Markert CD, Bharadwaj S, Zhang Y, Childers MK, Furth ME.

J Immunol Methods. 2010 Jan 31;352(1-2):174-7. doi: 10.1016/j.jim.2009.11.011. Epub 2009 Nov 17.


Mesenchymal stem cells: emerging therapy for Duchenne muscular dystrophy.

Markert CD, Atala A, Cann JK, Christ G, Furth M, Ambrosio F, Childers MK.

PM R. 2009 Jun;1(6):547-59. doi: 10.1016/j.pmrj.2009.02.013. Review.


Assessment: Botulinum neurotoxin in the treatment of autonomic disorders and pain (an evidence-based review): report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology.

Naumann M, So Y, Argoff CE, Childers MK, Dykstra DD, Gronseth GS, Jabbari B, Kaufmann HC, Schurch B, Silberstein SD, Simpson DM; Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology.

Neurology. 2008 May 6;70(19):1707-14. doi: 10.1212/01.wnl.0000311390.87642.d8.


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