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Items: 24

1.

Personalized gene and cell therapy for Duchenne Muscular Dystrophy.

Barthélémy F, Wein N.

Neuromuscul Disord. 2018 Oct;28(10):803-824. doi: 10.1016/j.nmd.2018.06.009. Epub 2018 Jul 26. Review.

PMID:
30224293
2.

Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach.

Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM.

J Neuromuscul Dis. 2017;4(3):199-207. doi: 10.3233/JND-170233.

PMID:
28869484
3.

Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells.

Massouridès E, Polentes J, Mangeot PE, Mournetas V, Nectoux J, Deburgrave N, Nusbaum P, Leturcq F, Popplewell L, Dickson G, Wein N, Flanigan KM, Peschanski M, Chelly J, Pinset C.

Skelet Muscle. 2015 Nov 14;5:40. doi: 10.1186/s13395-015-0062-6. eCollection 2015.

4.

The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development.

Vulin A, Wein N, Simmons TR, Rutherford AM, Findlay AR, Yurkoski JA, Kaminoh Y, Flanigan KM.

Neuromuscul Disord. 2015 Nov;25(11):827-34. doi: 10.1016/j.nmd.2015.08.005. Epub 2015 Aug 11.

PMID:
26365037
5.

Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients' Cells.

Barthélémy F, Blouin C, Wein N, Mouly V, Courrier S, Dionnet E, Kergourlay V, Mathieu Y, Garcia L, Butler-Browne G, Lamaze C, Lévy N, Krahn M, Bartoli M.

J Neuromuscul Dis. 2015 Sep 2;2(3):281-290.

6.

Genetics and emerging treatments for Duchenne and Becker muscular dystrophy.

Wein N, Alfano L, Flanigan KM.

Pediatr Clin North Am. 2015 Jun;62(3):723-42. doi: 10.1016/j.pcl.2015.03.008. Epub 2015 Apr 20. Review.

PMID:
26022172
7.

Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Brioschi S, Bovolenta M, Neri M, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2015 May;21(5):537. doi: 10.1038/nm0515-537c. No abstract available.

PMID:
25951531
8.

Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2015 Apr;21(4):414. doi: 10.1038/nm0415-414b. No abstract available.

PMID:
25849273
9.

Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.

Findlay AR, Wein N, Kaminoh Y, Taylor LE, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Howard MT, Day JW, McDonald C, Nicolas A, Le Rumeur E, Weiss RB, Flanigan KM; United Dystrophinopathy Project.

Ann Neurol. 2015 Apr;77(4):668-74. doi: 10.1002/ana.24365. Epub 2015 Mar 2.

10.

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Brioschi S, Bovolenta M, Neri M, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2014 Sep;20(9):992-1000. doi: 10.1038/nm.3628. Epub 2014 Aug 10. Erratum in: Nat Med. 2015 Apr;21(4):414. Nat Med. 2015 May;21(5):537. Brioschi, Simona [added]; Bovolenta, Matteo [added]; Neri, Marcella [added].

11.

The ZZ domain of dystrophin in DMD: making sense of missense mutations.

Vulin A, Wein N, Strandjord DM, Johnson EK, Findlay AR, Maiti B, Howard MT, Kaminoh YJ, Taylor LE, Simmons TR, Ray WC, Montanaro F, Ervasti JM, Flanigan KM.

Hum Mutat. 2014 Feb;35(2):257-64. doi: 10.1002/humu.22479. Epub 2013 Dec 2.

12.

Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells.

Meregalli M, Navarro C, Sitzia C, Farini A, Montani E, Wein N, Razini P, Beley C, Cassinelli L, Parolini D, Belicchi M, Parazzoli D, Garcia L, Torrente Y.

FEBS J. 2013 Dec;280(23):6045-60. doi: 10.1111/febs.12523. Epub 2013 Oct 8.

13.

Cigarette smoking and measures of impulsivity in a college sample.

Balevich EC, Wein ND, Flory JD.

Subst Abus. 2013;34(3):256-62. doi: 10.1080/08897077.2012.763082.

14.

Risk of cardiovascular disease from antiretroviral therapy for HIV: a systematic review.

Bavinger C, Bendavid E, Niehaus K, Olshen RA, Olkin I, Sundaram V, Wein N, Holodniy M, Hou N, Owens DK, Desai M.

PLoS One. 2013;8(3):e59551. doi: 10.1371/journal.pone.0059551. Epub 2013 Mar 26. Review.

15.

Becker muscular dystrophy with widespread muscle hypertrophy and a non-sense mutation of exon 2.

Flanigan KM, Wein N, Gurvich OL, Howard MT, Weiss RB.

Neuromuscul Disord. 2013 Feb;23(2):192. doi: 10.1016/j.nmd.2012.11.012. Epub 2013 Jan 29. No abstract available.

PMID:
23369578
16.

UMD-DYSF, a novel locus specific database for the compilation and interactive analysis of mutations in the dysferlin gene.

Blandin G, Beroud C, Labelle V, Nguyen K, Wein N, Hamroun D, Williams B, Monnier N, Rufibach LE, Urtizberea JA, Cau P, Bartoli M, Lévy N, Krahn M.

Hum Mutat. 2012 Mar;33(3):E2317-31. doi: 10.1002/humu.22015. Epub 2011 Dec 29.

PMID:
22213072
17.

Validation of comparative genomic hybridization arrays for the detection of genomic rearrangements of the calpain-3 and dysferlin genes.

Bartoli M, Nègre P, Wein N, Bourgeois P, Pécheux C, Lévy N, Krahn M.

Clin Genet. 2012 Jan;81(1):99-101. doi: 10.1111/j.1399-0004.2011.01708.x. No abstract available.

PMID:
22150418
18.

Translational research and therapeutic perspectives in dysferlinopathies.

Barthélémy F, Wein N, Krahn M, Lévy N, Bartoli M.

Mol Med. 2011 Sep-Oct;17(9-10):875-82. doi: 10.2119/molmed.2011.00084. Epub 2011 May 6. Review.

19.

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.

20.

Therapeutic exon 'switching' for dysferlinopathies?

Lévy N, Wein N, Barthelemy F, Mouly V, Garcia L, Krahn M, Bartoli M.

Eur J Hum Genet. 2010 Sep;18(9):969-70; author reply 971. doi: 10.1038/ejhg.2010.73. Epub 2010 May 26. No abstract available.

21.

Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping.

Wein N, Avril A, Bartoli M, Beley C, Chaouch S, Laforêt P, Behin A, Butler-Browne G, Mouly V, Krahn M, Garcia L, Lévy N.

Hum Mutat. 2010 Feb;31(2):136-42. doi: 10.1002/humu.21160.

PMID:
19953532
22.

Immunolabelling and flow cytometry as new tools to explore dysferlinopathies.

Wein N, Krahn M, Courrier S, Bartoli M, Salort-Campana E, Nguyen K, Fernandez C, Pouget J, Fossat C, Depetris D, Leturcq F, Cau P, Levy N.

Neuromuscul Disord. 2010 Jan;20(1):57-60. doi: 10.1016/j.nmd.2009.08.004. Epub 2009 Oct 23.

PMID:
19854055
23.

Identification of different genomic deletions and one duplication in the dysferlin gene using multiplex ligation-dependent probe amplification and genomic quantitative PCR.

Krahn M, Borges A, Navarro C, Schuit R, Stojkovic T, Torrente Y, Wein N, Pécheux C, Lévy N.

Genet Test Mol Biomarkers. 2009 Aug;13(4):439-42. doi: 10.1089/gtmb.2009.0010.

PMID:
19594366
24.

[Effect of the unclamped umbilical cord on the feto-maternal microtransfusion].

Wein N, Schneider I.

Zentralbl Gynakol. 1970 Aug 22;92(34):1113-7. German. No abstract available.

PMID:
4995979

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