A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model

Molecular Therapy

Volumn 25, Issue 9, 2017, Pages 2038-2052

  Biferi, Maria Grazia ^a   Cohen Tannoudji, Mathilde ^a   Cappelletto, Ambra ^a   Giroux, Benoit ^a   Roda, Marianne ^a   Astord, Stéphanie ^a   Marais, Thibaut ^a   Bos, Corinne ^a   Voit, Thomas ^c   Ferry, Arnaud ^a,b   Barkats, Martine ^a  

^a SORBONNE UNIVERSITÉ   (France)

^b UNIVERSITÉ PARIS DESCARTES   (France)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

AAV; ALS; exon skipping; gene therapy; hSOD1

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ANTISENSE OLIGONUCLEOTIDE; COPPER ZINC SUPEROXIDE DISMUTASE; MESSENGER RNA; MESSENGER RNA PRECURSOR; SMALL NUCLEAR RNA;

ADULT; AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; CONVALESCENCE; DRUG EFFICACY; EXON SKIPPING; EXTENSOR DIGITORUM LONGUS MUSCLE; GASTROCNEMIUS MUSCLE; GENE CASSETTE; GENE DELETION; GENE EXPRESSION; GENE SILENCING; GENETIC VARIABILITY; INTRON; LIFE EXPECTANCY; MOTOR COORDINATION; MOUSE; MOUSE MODEL; MUSCLE DENERVATION; MUSCLE STRENGTH; NEWBORN; NONHUMAN; STOP CODON; SURVIVAL; TIBIALIS ANTERIOR MUSCLE; VIRAL GENE THERAPY; WEIGHT REDUCTION; WILD TYPE; ADMINISTRATION AND DOSAGE; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; EXON; GENE ORDER; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HUMAN; METABOLISM; MORTALITY; MOTOR ACTIVITY; ONSET AGE; PATHOPHYSIOLOGY; PROCEDURES; RNA SPLICE SITE; SURVIVAL RATE; TRANSGENIC MOUSE;

AGE OF ONSET; AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; EXONS; GENE ORDER; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, TRANSGENIC; MOTOR ACTIVITY; OLIGONUCLEOTIDES, ANTISENSE; RECOVERY OF FUNCTION; RNA SPLICE SITES; RNA, MESSENGER; SUPEROXIDE DISMUTASE-1; SURVIVAL RATE; TRANSDUCTION, GENETIC;

EID: 85021066962     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.05.017     Document Type: Article

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