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Dystrophin immunity in Duchenne's muscular dystrophy.

Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM.

N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228.


Autoimmunity in a genetic disease—a cautionary tale.

Moore MJ, Flotte TR.

N Engl J Med. 2010 Oct 7;363(15):1473-5. doi: 10.1056/NEJMe1009056. No abstract available.


Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect.

Flanigan KM, Campbell K, Viollet L, Wang W, Gomez AM, Walker CM, Mendell JR.

Hum Gene Ther. 2013 Sep;24(9):797-806. doi: 10.1089/hum.2013.092.


Immune response to full-length dystrophin delivered to Dmd muscle by a high-capacity adenoviral vector.

Gilchrist SC, Ontell MP, Kochanek S, Clemens PR.

Mol Ther. 2002 Sep;6(3):359-68.


Dystrophin delivery in dystrophin-deficient DMDmdx skeletal muscle by isogenic muscle-derived stem cell transplantation.

Ikezawa M, Cao B, Qu Z, Peng H, Xiao X, Pruchnic R, Kimura S, Miike T, Huard J.

Hum Gene Ther. 2003 Nov 1;14(16):1535-46.


Gene therapy for Duchenne muscular dystrophy: AAV leads the way.

Judge LM, Chamberlain JS.

Acta Myol. 2005 Dec;24(3):184-93. Review.


Identification of an HLA-A*0201-restricted epitopic peptide from human dystrophin: application in duchenne muscular dystrophy gene therapy.

Ginhoux F, Doucet C, Leboeuf M, Lemonnier FA, Danos O, Davoust J, Firat H.

Mol Ther. 2003 Aug;8(2):274-83.


A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs.

Shin JH, Yue Y, Srivastava A, Smith B, Lai Y, Duan D.

Hum Gene Ther. 2012 Feb;23(2):202-9. doi: 10.1089/hum.2011.147. Epub 2011 Dec 14.


Myoblast transfer in the treatment of Duchenne's muscular dystrophy.

Mendell JR, Kissel JT, Amato AA, King W, Signore L, Prior TW, Sahenk Z, Benson S, McAndrew PE, Rice R, et al.

N Engl J Med. 1995 Sep 28;333(13):832-8.


Sustained muscle expression of dystrophin from a high-capacity adenoviral vector with systemic gene transfer of T cell costimulatory blockade.

Jiang Z, Schiedner G, van Rooijen N, Liu CC, Kochanek S, Clemens PR.

Mol Ther. 2004 Oct;10(4):688-96.


CTLA4Ig delivered by high-capacity adenoviral vector induces stable expression of dystrophin in mdx mouse muscle.

Jiang Z, Schiedner G, Gilchrist SC, Kochanek S, Clemens PR.

Gene Ther. 2004 Oct;11(19):1453-61.


Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC.

N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. Erratum in: N Engl J Med. 2011 Oct 6;365(14):1361.


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.


The CRISPR Way to Think about Duchenne's.

Calos MP.

N Engl J Med. 2016 Apr 28;374(17):1684-6. doi: 10.1056/NEJMcibr1601383. No abstract available.


Immunological hurdles in the path to gene therapy for Duchenne muscular dystrophy.

Wells DJ, Ferrer A, Wells KE.

Expert Rev Mol Med. 2002 Nov 4;4(23):1-23. Review.


MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy.

Barbash IM, Cecchini S, Faranesh AZ, Virag T, Li L, Yang Y, Hoyt RF, Kornegay JN, Bogan JR, Garcia L, Lederman RJ, Kotin RM.

Gene Ther. 2013 Mar;20(3):274-82. doi: 10.1038/gt.2012.38. Epub 2012 May 3.


Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.

Koo T, Malerba A, Athanasopoulos T, Trollet C, Boldrin L, Ferry A, Popplewell L, Foster H, Foster K, Dickson G.

Hum Gene Ther. 2011 Nov;22(11):1379-88. doi: 10.1089/hum.2011.020. Epub 2011 May 25.


Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy.

Wang Z, Allen JM, Riddell SR, Gregorevic P, Storb R, Tapscott SJ, Chamberlain JS, Kuhr CS.

Hum Gene Ther. 2007 Jan;18(1):18-26.


Restoration of all dystrophin protein interactions by functional domains in trans does not rescue dystrophy.

Gardner KL, Kearney JA, Edwards JD, Rafael-Fortney JA.

Gene Ther. 2006 May;13(9):744-51.


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