RNA splicing manipulation: Strategies to modify gene expression for a variety of therapeutic outcomes

Current Gene Therapy

Volumn 11, Issue 4, 2011, Pages 259-275

  Wilton, Steve D ^a   Fletcher, Susan ^a  

^a UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

Alternative splicing; Complex disorders; Duchenne muscular dystrophy; Inherited disease; Isoform; Mutation suppression; Rna; Spliceosome

Indexed keywords

2' O (2 METHOXY) ETHYL OLIGOMER DERIVATIVE; 2' O (2 METHOXY) ETHYL PHOSPHOROTHIOATE MODIFIED ANTISENSE OLIGONUCLEOTIDE; 2' O METHYL PHOSPHOROTHIOATE OLIGOMER DERIVATIVE; ANTISENSE OLIGODEOXYNUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE; APC PROTEIN; BETA GLOBIN; BRCA1 PROTEIN; DYSTROPHIN; ETEPLIRSEN; GENE THERAPY AGENT; MESSENGER RNA; MICRORNA; OLIGOMER; PEPTIDE NUCLEIC ACID; PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER DERIVATIVE; PRO 051; PROTEIN MLH1; PROTEIN MSH2; RIBONUCLEASE H; RNA; RNA BINDING PROTEIN; RNA DERIVATIVE; RNA DIRECTED DNA POLYMERASE; SMALL INTERFERING RNA; SURVIVAL MOTOR NEURON PROTEIN; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG; UNINDEXED DRUG; UNTRANSLATED RNA; VIRUS PROTEIN;

BETA THALASSEMIA; CANCER SUSCEPTIBILITY; CHICK; CHROMATIN STRUCTURE; CLINICAL TRIAL (TOPIC); CONJUGATE; CROHN DISEASE; DISEASE COURSE; DISEASE SEVERITY; DOWN REGULATION; DRUG EFFICACY; DRUG MEGADOSE; DRUG POTENCY; DRUG SAFETY; DRUG STRUCTURE; DRUG TOLERABILITY; DRUG UPTAKE; DUCHENNE MUSCULAR DYSTROPHY; ENZYME ACTIVITY; ENZYME DEGRADATION; ENZYME STABILITY; EUKARYOTE; EXON; EXON SKIPPING; FIBROBLAST; FRAMESHIFT MUTATION; GENE CONTROL; GENE EXPRESSION; GENE REPRESSION; GENE SILENCING; GENETIC MANIPULATION; GENETIC PREDISPOSITION; GENETIC VARIABILITY; GENOME; HUMAN; HUMAN GENOME; HUMAN GENOME PROJECT; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; INTRON; MISSENSE MUTATION; MORTALITY; NEOPLASM; NONHUMAN; PROTEIN MODIFICATION; PROTEIN TARGETING; REVIEW; RNA BINDING; RNA SPLICING; RNA TRANSLATION; SCHIZOPHRENIA; SINGLE DRUG DOSE; SINGLE NUCLEOTIDE POLYMORPHISM; SPINAL MUSCULAR ATROPHY; SPLICEOSOME; TRANSCRIPTION REGULATION; TREATMENT OUTCOME; TUMOR SUPPRESSOR GENE; VIRUS INFECTION; WHEAT GERM; WILD TYPE; X CHROMOSOME;

ALTERNATIVE SPLICING; ANIMALS; BETA-THALASSEMIA; CLINICAL TRIALS, PHASE I AS TOPIC; DOWN-REGULATION; EXONS; FRAMESHIFT MUTATION; GENE EXPRESSION; HUMANS; INTRONS; MUSCULAR ATROPHY, SPINAL; MUSCULAR DYSTROPHY, DUCHENNE; NEOPLASMS; OLIGONUCLEOTIDES, ANTISENSE; RNA PRECURSORS; TREATMENT OUTCOME;

EUKARYOTA;

EID: 79959975621     PISSN: 15665232     EISSN: 18755631     Source Type: Journal    
DOI: 10.2174/156652311796150381     Document Type: Review

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