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List of works by Stéphanie Lorain

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

scientific article published on 11 June 2013

Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy

scientific article

Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy

scientific article published on 15 December 2017

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

scientific article published on 4 July 2016

Core histones and HIRIP3, a novel histone-binding protein, directly interact with WD repeat protein HIRA

scientific article

Correction of the Middle Eastern M712T mutation causing GNE myopathy by trans-splicing

scientific article published on 22 November 2013

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

scientific article

Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites

scientific article published on 22 April 2015

Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy.

scientific article published on 8 June 2016

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches

scientific article published on 16 July 2013

Exon exchange approach to repair Duchenne dystrophin transcripts

scientific article

HIRA, a mammalian homologue of Saccharomyces cerevisiae transcriptional co-repressors, interacts with Pax3

scientific article

Identification of human and mouse HIRA-interacting protein-5 (HIRIP5), two mammalian representatives in a family of phylogenetically conserved proteins with a role in the biogenesis of Fe/S proteins

scientific article

Intradermal Immunization with rAAV1 Vector Induces Robust Memory CD8(+) T Cell Responses Independently of Transgene Expression in DCs.

scientific article

Intrinsic transgene immunogenicity gears CD8(+) T-cell priming after rAAV-mediated muscle gene transfer.

scientific article

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

scientific article published on 11 September 2012

Repair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy

scientific article

Repair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa

scientific article published on 26 January 2015

Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing

scientific article published on 13 September 2016

Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice

scientific article

Retinoblastoma protein represses transcription by recruiting a histone deacetylase

scientific article

Structural Organization of the WD repeat protein-encoding gene HIRA in the DiGeorge syndrome critical region of human chromosome 22.

scientific article published in January 1996

Subnuclear localization and mitotic phosphorylation of HIRA, the human homologue of Saccharomyces cerevisiae transcriptional regulators Hir1p/Hir2p

scientific article published on September 2001

The HIR protein family: isolation and characterization of a complete murine cDNA.

scientific article published on April 1996

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

scientific article

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