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Items: 11


Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy.

Wasala NB, Yue Y, Lostal W, Wasala LP, Niranjan N, Hajjar RJ, Babu GJ, Duan D.

Mol Ther. 2020 Jan 10. pii: S1525-0016(20)30006-X. doi: 10.1016/j.ymthe.2019.12.011. [Epub ahead of print]


Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A.

Lostal W, Roudaut C, Faivre M, Charton K, Suel L, Bourg N, Best H, Smith JE, Gohlke J, Corre G, Li X, Elbeck Z, Knöll R, Deschamps JY, Granzier H, Richard I.

Sci Transl Med. 2019 Nov 27;11(520). pii: eaat6072. doi: 10.1126/scitranslmed.aat6072.


In Vivo Monitoring of Calpain Activity by Forster Resonance Energy Transfer.

Lostal W, Stockholm D, Richard I.

Methods Mol Biol. 2019;1915:57-66. doi: 10.1007/978-1-4939-8988-1_5.


233rd ENMC International Workshop:: Clinical Trial Readiness for Calpainopathies, Naarden, The Netherlands, 15-17 September 2017.

Lostal W, Urtizberea JA, Richard I; calpain 3 study group.

Neuromuscul Disord. 2018 Jun;28(6):540-549. doi: 10.1016/j.nmd.2018.03.010. Epub 2018 Mar 28. No abstract available.


Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies.

Israeli D, Poupiot J, Amor F, Charton K, Lostal W, Jeanson-Leh L, Richard I.

Sci Rep. 2016 Jun 21;6:28097. doi: 10.1038/srep28097.


A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence.

Pryadkina M, Lostal W, Bourg N, Charton K, Roudaut C, Hirsch ML, Richard I.

Mol Ther Methods Clin Dev. 2015 Mar 25;2:15009. doi: 10.1038/mtm.2015.9. eCollection 2015.


Full-length dystrophin reconstitution with adeno-associated viral vectors.

Lostal W, Kodippili K, Yue Y, Duan D.

Hum Gene Ther. 2014 Jun;25(6):552-62. doi: 10.1089/hum.2013.210. Epub 2014 Mar 31.


Long-term robust myocardial transduction of the dog heart from a peripheral vein by adeno-associated virus serotype-8.

Pan X, Yue Y, Zhang K, Lostal W, Shin JH, Duan D.

Hum Gene Ther. 2013 Jun;24(6):584-94. doi: 10.1089/hum.2013.044. Epub 2013 May 9.


Lack of correlation between outcomes of membrane repair assay and correction of dystrophic changes in experimental therapeutic strategy in dysferlinopathy.

Lostal W, Bartoli M, Roudaut C, Bourg N, Krahn M, Pryadkina M, Borel P, Suel L, Roche JA, Stockholm D, Bloch RJ, Levy N, Bashir R, Richard I.

PLoS One. 2012;7(5):e38036. doi: 10.1371/journal.pone.0038036. Epub 2012 May 29.


A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy.

Krahn M, Wein N, Bartoli M, Lostal W, Courrier S, Bourg-Alibert N, Nguyen K, Vial C, Streichenberger N, Labelle V, DePetris D, Pécheux C, Leturcq F, Cau P, Richard I, Lévy N.

Sci Transl Med. 2010 Sep 22;2(50):50ra69. doi: 10.1126/scitranslmed.3000951.


Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer.

Lostal W, Bartoli M, Bourg N, Roudaut C, Bentaïb A, Miyake K, Guerchet N, Fougerousse F, McNeil P, Richard I.

Hum Mol Genet. 2010 May 15;19(10):1897-907. doi: 10.1093/hmg/ddq065. Epub 2010 Feb 13.


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