Splicing modulation therapy in the treatment of genetic diseases

Application of Clinical Genetics

Volumn 7, Issue , 2014, Pages 245-252

  Arechavala Gomeza, Virginia ^a   Khoo, Bernard ^b   Aartsma Rus, Annemieke ^c  

^a BIOCRUCES BIZKAIA HEALTH RESEARCH INSTITUTE   (Spain)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Alternative splicing; Antisense oligonucleotides; Cryptic splicing; Splicing; Therapy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; HEMOGLOBIN BETA CHAIN; MEPROBAMATE; MESSENGER RNA; STAT3 PROTEIN; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2;

ALTERNATIVE RNA SPLICING; APOB GENE; APOPTOSIS; ARTICLE; BECKER MUSCULAR DYSTROPHY; BETA THALASSEMIA; CODON; DISEASE COURSE; DMD GENE; EXON; GENE; GENE DELETION; GENE DUPLICATION; GENE EXPRESSION; GENE MUTATION; GENE TARGETING; GENE THERAPY; GENETIC DISORDER; INTRON; LMNA GENE; NONHUMAN; OPEN READING FRAME; POINT MUTATION; PROTEIN EXPRESSION; RNA EDITING; RNA INTERFERENCE; RNA TRANSLATION; SPLICEOSOME; USH1C GENE;

EID: 84937547072     PISSN: None     EISSN: 1178704X     Source Type: Journal    
DOI: 10.2147/TACG.S71506     Document Type: Article

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