Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

Biochimica et Biophysica Acta - Biomembranes

Volumn 1798, Issue 12, 2010, Pages 2296-2303

  Moulton, Hong M ^a   Moulton, Jon D ^b  

^a OREGON STATE UNIVERSITY   (United States)

^b Gene Tools LLC   (United States)

Author keywords

Cell penetrating peptide; Duchenne muscular dystrophy; Exon skipping; Morpholino; PMO; PPMO

Indexed keywords

(RXR)4 PEPTIDE; (RXRRBR)2 PEPTIDE; ASSLNIAX PEPTIDE; AVI 4658; CELL PENETRATING PEPTIDE; DYSTROPHIN; OLIGOMER; PHOSPHODIAMIDATE MORPHOLINO OLIGOMER; PHOSPHODIAMIDATE MORPHOLINO OLIGOMER E23; UNCLASSIFIED DRUG;

ANIMAL MUSCULAR DYSTROPHY; CARDIOMYOPATHY; CELL TRANSPORT; CLINICAL TRIAL; DIASTOLIC DYSFUNCTION; DOSAGE SCHEDULE COMPARISON; DOSE RESPONSE; DRUG DELIVERY SYSTEM; DRUG DOSE COMPARISON; DRUG DOSE REGIMEN; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG SPECIFICITY; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; HEART HYPERTROPHY; HEART MUSCLE CELL; HUMAN; KIDNEY CLEARANCE; LOW DRUG DOSE; MULTIPLE CYCLE TREATMENT; MUSCLE CELL; MUSCLE FUNCTION; NONHUMAN; PRIORITY JOURNAL; REPEATED DRUG DOSE; REVIEW; SINGLE DRUG DOSE; SKELETAL MUSCLE; SYSTEMIC THERAPY;

ANIMALS; CELL-PENETRATING PEPTIDES; CLINICAL TRIALS AS TOPIC; DOGS; DRUG DELIVERY SYSTEMS; DYSTROPHIN; HUMANS; MICE; MICE, INBRED MDX; MICE, KNOCKOUT; MORPHOLINES; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MYOCARDIUM; OLIGONUCLEOTIDES; RECOVERY OF FUNCTION; RNA SPLICING;

ANIMALIA; CANIS FAMILIARIS; MUS;

EID: 77958152873     PISSN: 00052736     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.bbamem.2010.02.012     Document Type: Review

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