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

Molecular Therapy

Volumn 23, Issue 5, 2015, Pages 918-930

  Berger, Adeline ^a   Lorain, Stéphanie ^a   Joséphine, Charlène ^b,c   Desrosiers, Melissa ^a   Peccate, Cécile ^a   Voit, Thomas ^a   Garcia, Luis ^d   Sahel, José Alain ^a,e,f,g   Bemelmans, Alexis Pierre ^a,b,c  

^a SORBONNE UNIVERSITÉ   (France)

^b INSTITUT DE RADIOPROTECTION ET DE SÛRETÉ NUCLÉAIRE   (France)

^c UNIVERSITÉ PARIS SACLAY   (France)

^d LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^e CENTRE HOSPITALIER NATIONAL D OPHTALMOLOGIE DES QUINZE VINGTS   (France)

^f FONDATION OPHTALMOLOGIQUE ADOLPHE DE ROTHSCHILD   (France)

^g UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

MESSENGER RNA; PARVOVIRUS VECTOR; RHODOPSIN; RNA PRECURSOR;

3' UNTRANSLATED REGION; 5' UNTRANSLATED REGION; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; AUTOSOMAL DOMINANT RETINITIS PIGMENTOSA; BINDING SITE; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DISEASE SEVERITY; DRUG EFFICACY; EMBRYO; EXON; FEASIBILITY STUDY; GENE EXPRESSION; GENE MUTATION; GENE TARGETING; GENETIC TRANSFECTION; HEK293 CELL LINE; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; INTERNALIZATION; INTRON; MOUSE; NONHUMAN; PATHOGENESIS; PHENOTYPE; PHOTORECEPTOR; PROTEIN ENGINEERING; PROTEIN LOCALIZATION; PROTEIN PROCESSING; RETINITIS PIGMENTOSA; RHO GENE; RNA REPAIR; SPLICEOSOME MEDIATED RNA TRANS SPLICING; TRANS SPLICING; TRANSCRIPTION INITIATION SITE; VIRAL GENE DELIVERY SYSTEM; WILD TYPE; ANIMAL; CHEMISTRY; DOMINANT GENE; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; METABOLISM; PHOTORECEPTOR CELL; PROTEIN DOMAIN; PROTEIN TRANSPORT; RNA SPLICING; TRANSFORMED CELL LINE; TRANSGENIC MOUSE;

ANIMALS; BINDING SITES; CELL LINE, TRANSFORMED; GENE EXPRESSION; GENES, DOMINANT; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INTRONS; MICE; MICE, TRANSGENIC; PHENOTYPE; PHOTORECEPTOR CELLS; PROTEIN INTERACTION DOMAINS AND MOTIFS; PROTEIN TRANSPORT; RETINITIS PIGMENTOSA; RHODOPSIN; RNA PRECURSORS; RNA SPLICING; RNA, MESSENGER; TRANS-SPLICING; TRANSDUCTION, GENETIC;

EID: 84929047520     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.11     Document Type: Article

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