Splicing therapeutics in SMN2 and APOB

Current Opinion in Molecular Therapeutics

Volumn 11, Issue 2, 2009, Pages 108-115

  Khoo, Bernard ^a   Krainer, Adrian R ^b  

^a ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

^b Howard Hughes Medical Institute Cold Spring Harbor Laboratory Cold Spring Harbor   (United States)

Author keywords

Antisense oligonucleotide; APOB; Apolipoprotein B; Cholesterol; RNA splicing; SMN2; Spinal muscular atrophy

Indexed keywords

ACLARUBICIN; ANTISENSE OLIGODEOXYNUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B; APOLIPOPROTEIN B100; APOLIPOPROTEIN B48; APOLIPOPROTEIN E; ARGININE; CHOLESTEROL; CHYLOMICRON; INDOPROFEN; INTERMEDIATE DENSITY LIPOPROTEIN; LIPOPROTEIN A; LOW DENSITY LIPOPROTEIN; MESSENGER RNA; MESSENGER RNA PRECURSOR; PEPTIDE NUCLEIC ACID; SERINE; SMAD PROTEIN; SMALL NUCLEAR RIBONUCLEOPROTEIN; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2; VERY LOW DENSITY LIPOPROTEIN;

3' UNTRANSLATED REGION; 5' UNTRANSLATED REGION; ANTISENSE THERAPY; CARBOXY TERMINAL SEQUENCE; CELL STRAIN HEPG2; CHOLESTEROL BLOOD LEVEL; DRUG TARGETING; EXON; GENE DELETION; GENE LOCATION; GENE STRUCTURE; GENE THERAPY; GENETIC ENGINEERING; GENETIC ENGINEERING AND GENE TECHNOLOGY; HUMAN; INTRON; OUTCOME ASSESSMENT; POINT MUTATION; PROTEIN DEGRADATION; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PROTEIN TARGETING; REVIEW; RNA SPLICING; SPINAL MUSCULAR ATROPHY; TRANS SPLICING;

ANTICHOLESTEREMIC AGENTS; APOLIPOPROTEINS B; EXONS; HUMANS; MODELS, BIOLOGICAL; MUSCULAR ATROPHY, SPINAL; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING; SMN COMPLEX PROTEINS;

EID: 63849196603     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

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