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Items: 41


Exon Skipping in a Dysf-Missense Mutant Mouse Model.

Malcher J, Heidt L, Goyenvalle A, Escobar H, Marg A, Beley C, Benchaouir R, Bader M, Spuler S, García L, Schöwel V.

Mol Ther Nucleic Acids. 2018 Dec 7;13:198-207. doi: 10.1016/j.omtn.2018.08.013. Epub 2018 Aug 22.


Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping.

Relizani K, Goyenvalle A.

Methods Mol Biol. 2018;1828:381-394. doi: 10.1007/978-1-4939-8651-4_24.


Exon-skipping advances for Duchenne muscular dystrophy.

Echevarría L, Aupy P, Goyenvalle A.

Hum Mol Genet. 2018 Aug 1;27(R2):R163-R172. doi: 10.1093/hmg/ddy171.


The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders.

Aupy P, Echevarría L, Relizani K, Goyenvalle A.

Biomedicines. 2017 Dec 22;6(1). pii: E2. doi: 10.3390/biomedicines6010002. Review.


Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping.

Omairi S, Hau KL, Collin-Hooper H, Montanaro F, Goyenvalle A, Garcia L, Patel K.

Mol Ther Nucleic Acids. 2017 Dec 15;9:409-418. doi: 10.1016/j.omtn.2017.10.014. Epub 2017 Oct 26.


The Use of Antisense Oligonucleotides for the Treatment of Duchenne Muscular Dystrophy.

Relizani K, Goyenvalle A.

Methods Mol Biol. 2018;1687:171-183. doi: 10.1007/978-1-4939-7374-3_12.


Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model.

Relizani K, Griffith G, Echevarría L, Zarrouki F, Facchinetti P, Vaillend C, Leumann C, Garcia L, Goyenvalle A.

Mol Ther Nucleic Acids. 2017 Sep 15;8:144-157. doi: 10.1016/j.omtn.2017.06.013. Epub 2017 Jun 22.


Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment.

Robin V, Griffith G, Carter JL, Leumann CJ, Garcia L, Goyenvalle A.

Mol Ther Nucleic Acids. 2017 Jun 16;7:81-89. doi: 10.1016/j.omtn.2017.02.009. Epub 2017 Mar 14.


Delivery is key: lessons learnt from developing splice-switching antisense therapies.

Godfrey C, Desviat LR, Smedsrød B, Piétri-Rouxel F, Denti MA, Disterer P, Lorain S, Nogales-Gadea G, Sardone V, Anwar R, El Andaloussi S, Lehto T, Khoo B, Brolin C, van Roon-Mom WM, Goyenvalle A, Aartsma-Rus A, Arechavala-Gomeza V.

EMBO Mol Med. 2017 May;9(5):545-557. doi: 10.15252/emmm.201607199. Review.


Viral Vector-Mediated Antisense Therapy for Genetic Diseases.

Imbert M, Dias-Florencio G, Goyenvalle A.

Genes (Basel). 2017 Jan 26;8(2). pii: E51. doi: 10.3390/genes8020051. Review.


Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides.

Goyenvalle A, Leumann C, Garcia L.

J Neuromuscul Dis. 2016 May 27;3(2):157-167. Review.


Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.

Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A.

Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Review.


Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides.

Ezzat K, Aoki Y, Koo T, McClorey G, Benner L, Coenen-Stass A, O'Donovan L, Lehto T, Garcia-Guerra A, Nordin J, Saleh AF, Behlke M, Morris J, Goyenvalle A, Dugovic B, Leumann C, Gordon S, Gait MJ, El-Andaloussi S, Wood MJ.

Nano Lett. 2015 Jul 8;15(7):4364-73. doi: 10.1021/acs.nanolett.5b00490. Epub 2015 Jun 15.


Gene and splicing therapies for neuromuscular diseases.

Benchaouir R, Robin V, Goyenvalle A.

Front Biosci (Landmark Ed). 2015 Jun 1;20:1190-233. Review.


[Functional correction and cognitive improvement in dystrophic mice using splice-switching tricyclo-DNA oligomers].

Goyenvalle A, Griffith G, Avril A, Amthor H, Garcia L.

Med Sci (Paris). 2015 Mar;31(3):253-6. doi: 10.1051/medsci/20153103009. Epub 2015 Apr 8. French. No abstract available.


Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.

Goyenvalle A, Griffith G, Babbs A, El Andaloussi S, Ezzat K, Avril A, Dugovic B, Chaussenot R, Ferry A, Voit T, Amthor H, Bühr C, Schürch S, Wood MJ, Davies KE, Vaillend C, Leumann C, Garcia L.

Nat Med. 2015 Mar;21(3):270-5. doi: 10.1038/nm.3765. Epub 2015 Feb 2.


AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.

Le Hir M, Goyenvalle A, Peccate C, Précigout G, Davies KE, Voit T, Garcia L, Lorain S.

Mol Ther. 2013 Aug;21(8):1551-8. doi: 10.1038/mt.2013.121. Epub 2013 Jun 11.


The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.

Eckenfelder A, Tordo J, Babbs A, Davies KE, Goyenvalle A, Danos O.

Mol Ther Nucleic Acids. 2012 Jun 26;1:e31. doi: 10.1038/mtna.2012.24.


Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

Vulin A, Barthélémy I, Goyenvalle A, Thibaud JL, Beley C, Griffith G, Benchaouir R, le Hir M, Unterfinger Y, Lorain S, Dreyfus P, Voit T, Carlier P, Blot S, Garcia L.

Mol Ther. 2012 Nov;20(11):2120-33. doi: 10.1038/mt.2012.181. Epub 2012 Sep 11.


Therapeutic approaches to muscular dystrophies.

Goyenvalle A.

Curr Gene Ther. 2012 Jun;12(3):137-8. No abstract available.


Splicing modulation mediated by small nuclear RNAs as therapeutic approaches for muscular dystrophies.

Benchaouir R, Goyenvalle A.

Curr Gene Ther. 2012 Jun;12(3):179-91. Review.


Impaired adaptive response to mechanical overloading in dystrophic skeletal muscle.

Joanne P, Hourdé C, Ochala J, Caudéran Y, Medja F, Vignaud A, Mouisel E, Hadj-Said W, Arandel L, Garcia L, Goyenvalle A, Mounier R, Zibroba D, Sakamoto K, Butler-Browne G, Agbulut O, Ferry A.

PLoS One. 2012;7(4):e35346. doi: 10.1371/journal.pone.0035346. Epub 2012 Apr 12. Erratum in: PLoS One. 2013;8(6). doi:10.1371/annotation/2c0f8405-a2cc-4240-8e1b-117d3ac1a154. Sakamato, Kei [name corrected to Sakamoto, Kei].


Engineering U7snRNA gene to reframe transcripts.

Goyenvalle A.

Methods Mol Biol. 2012;867:259-71. doi: 10.1007/978-1-61779-767-5_17.


Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.

Goyenvalle A, Babbs A, Wright J, Wilkins V, Powell D, Garcia L, Davies KE.

Hum Mol Genet. 2012 Jun 1;21(11):2559-71. doi: 10.1093/hmg/dds082. Epub 2012 Mar 2.


Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.

Goyenvalle A, Wright J, Babbs A, Wilkins V, Garcia L, Davies KE.

Mol Ther. 2012 Jun;20(6):1212-21. doi: 10.1038/mt.2012.26. Epub 2012 Feb 21.


Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy.

Goyenvalle A, Davies KE.

Skelet Muscle. 2011 Feb 9;1(1):8. doi: 10.1186/2044-5040-1-8.


Therapeutic approaches to muscular dystrophy.

Goyenvalle A, Seto JT, Davies KE, Chamberlain J.

Hum Mol Genet. 2011 Apr 15;20(R1):R69-78. doi: 10.1093/hmg/ddr105. Epub 2011 Mar 24. Review.


Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.

Goyenvalle A, Davies KE.

Methods Mol Biol. 2011;709:179-96. doi: 10.1007/978-1-61737-982-6_11.


Diaphragm rescue alone prevents heart dysfunction in dystrophic mice.

Crisp A, Yin H, Goyenvalle A, Betts C, Moulton HM, Seow Y, Babbs A, Merritt T, Saleh AF, Gait MJ, Stuckey DJ, Clarke K, Davies KE, Wood MJ.

Hum Mol Genet. 2011 Feb 1;20(3):413-21. doi: 10.1093/hmg/ddq477. Epub 2010 Nov 9.


Rescue of a dystrophin-like protein by exon skipping in vivo restores GABAA-receptor clustering in the hippocampus of the mdx mouse.

Vaillend C, Perronnet C, Ros C, Gruszczynski C, Goyenvalle A, Laroche S, Danos O, Garcia L, Peltekian E.

Mol Ther. 2010 Sep;18(9):1683-8. doi: 10.1038/mt.2010.134. Epub 2010 Jun 29.


Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.

Goyenvalle A, Babbs A, Powell D, Kole R, Fletcher S, Wilton SD, Davies KE.

Mol Ther. 2010 Jan;18(1):198-205. doi: 10.1038/mt.2009.248. Epub 2009 Oct 20.


Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.

Goyenvalle A, Babbs A, van Ommen GJ, Garcia L, Davies KE.

Mol Ther. 2009 Jul;17(7):1234-40. doi: 10.1038/mt.2009.113. Epub 2009 May 19.


Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy cells.

Chaouch S, Mouly V, Goyenvalle A, Vulin A, Mamchaoui K, Negroni E, Di Santo J, Butler-Browne G, Torrente Y, Garcia L, Furling D.

Hum Gene Ther. 2009 Jul;20(7):784-90. doi: 10.1089/hum.2008.163.


Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice.

Benchaouir R, Meregalli M, Farini A, D'Antona G, Belicchi M, Goyenvalle A, Battistelli M, Bresolin N, Bottinelli R, Garcia L, Torrente Y.

Cell Stem Cell. 2007 Dec 13;1(6):646-57. doi: 10.1016/j.stem.2007.09.016.


[Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice].

Benchaouir R, Meregalli M, Farini A, D'Antona G, Belicchi M, Goyenvalle A, Battistelli M, Bresolin N, Bottinelli R, Garcia L, Torrente Y.

Med Sci (Paris). 2008 Jan;24(1):99-101. doi: 10.1051/medsci/200824199. French. No abstract available.


Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles.

Lorain S, Gross DA, Goyenvalle A, Danos O, Davoust J, Garcia L.

Mol Ther. 2008 Mar;16(3):541-7. doi: 10.1038/ Epub 2008 Jan 8.


Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy.

't Hoen PA, van der Wees CG, Aartsma-Rus A, Turk R, Goyenvalle A, Danos O, Garcia L, van Ommen GJ, den Dunnen JT, van Deutekom JC.

Pharmacogenomics. 2006 Apr;7(3):281-97.


The Journal of Gene Medicine European Society of Gene Therapy Young Investigator Award 2005.

Goyenvalle A.

J Gene Med. 2005 Sep;7(9):1257-8. No abstract available.


Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies.

Bartoli M, Poupiot J, Goyenvalle A, Perez N, Garcia L, Danos O, Richard I.

Gene Ther. 2006 Jan;13(1):20-8.


[An opening in Duchenne muscular dystrophy: persistent therapeutic rescue of dystrophin by vectorized antisense mediated exon skipping in mdx mice].

Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O.

Med Sci (Paris). 2004 Dec;20(12):1163-5. French. No abstract available.


Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.

Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O.

Science. 2004 Dec 3;306(5702):1796-9. Epub 2004 Nov 4.

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