Adeno-associated virus (AAV) mediated dystrophin gene transfer studies and exon skipping strategies for duchenne muscular dystrophy (DMD)

Current Gene Therapy

Volumn 15, Issue 4, 2015, Pages 395-415

  Kawecka, Klaudia ^a   Theodoulides, Michael ^a   Hasoglu, Yalin ^a   Jarmin, Susan ^b   Kymalainen, Hanna ^b   Le Heron, Anita ^b   Popplewell, Linda ^b   Malerba, Alberto ^b   Dickson, George ^b   Athanasopoulos, Takis ^a,b  

^a UNIVERSITY OF WOLVERHAMPTON   (United Kingdom)

^b UNIVERSITY OF LONDON   (United Kingdom)

Author keywords

Adeno associated virus (AAV); Antisense oligonucleotides (AONs); Duchenne muscular dystrophy (DMD); Dystrophin; Exon skipping; In vivo

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATALUREN; CD8 ANTIGEN; COMPLEMENTARY DNA; CRISPR ASSOCIATED PROTEIN; DRISAPERSEN; DYSTROPHIN; DYSTROPHIN ASSOCIATED PROTEIN COMPLEX; ETEPLIRSEN; GENOMIC DNA; NEURONAL NITRIC OXIDE SYNTHASE; PARVOVIRUS VECTOR; DMD PROTEIN, HUMAN; OXADIAZOLE DERIVATIVE;

ADENO ASSOCIATED VIRUS; ARTICLE; COST; DNA END JOINING REPAIR; DUCHENNE CARDIOMYOPATHY; DUCHENNE MUSCULAR DYSTROPHY; DYSTROPHIN GENE; EXON SKIPPING; FRAMESHIFT MUTATION; GENE; GENE DELETION; GENE TRANSFER; GENETIC TRANSFECTION; HEART MUSCLE; HUMAN; IMMUNE RESPONSE; IMMUNOGENICITY; NONHUMAN; OPEN READING FRAME; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PROTEIN STRUCTURE; RNA EDITING; SUPPLEMENTATION; TRANS SPLICING; TRANSCRIPTION REGULATION; UPTAKE ASSAY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRAL TROPISM; ADMINISTRATION AND DOSAGE; ANIMAL; CLINICAL TRIAL (TOPIC); DEFICIENCY; DEPENDOPARVOVIRUS; DISEASE MODEL; EXON; GENE THERAPY; GENE VECTOR; GENETICS; IMMUNOLOGY; MUSCULAR DYSTROPHY, DUCHENNE; PROCEDURES;

ADENO-ASSOCIATED VIRUS;

ANIMALS; CLINICAL TRIALS AS TOPIC; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DYSTROPHIN; EXONS; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; OXADIAZOLES; RNA EDITING;

EID: 84938678185     PISSN: 15665232     EISSN: 18755631     Source Type: Journal    
DOI: 10.2174/1566523215666150710123830     Document Type: Article

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