Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

Nucleic Acid Therapeutics

Volumn 27, Issue 3, 2017, Pages 130-143

  Shabanpoor, Fazel ^a   Hammond, Suzan M ^b   Abendroth, Frank ^a   Hazell, Gareth ^b   Wood, Matthew J A ^b   Gait, Michael J ^a  

^a MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

^b UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

Brain delivery; peptides; spinal muscular atrophy; splice switching oligonucleotide; survival motor neuron

Indexed keywords

ALANINE AMINOTRANSFERASE; AMINOCAPROIC ACID; APOLIPOPROTEIN E; ASPARTATE AMINOTRANSFERASE; CHOLINERGIC RECEPTOR; CREATININE; GANGLIOSIDE; GLYCOPROTEIN; LOW DENSITY LIPOPROTEIN RECEPTOR; LOW DENSITY LIPOPROTEIN RECEPTOR RELATED PROTEIN; MESSENGER RNA PRECURSOR; MORPHOLINO OLIGONUCLEOTIDE; PEPTIDE; PHOSPHORODIAMIDATE MORPHOLINO OLIGONUCLEOTIDE; PROTEGRIN; SURVIVAL MOTOR NEURON PROTEIN 2; SYNTHETIC PEPTIDE; TRANSFERRIN RECEPTOR; UNCLASSIFIED DRUG; UREA; BIOLOGICAL MARKER; NANOCONJUGATE; SMN2 PROTEIN, MOUSE;

ADULT; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; BLOOD BRAIN BARRIER; BODY WEIGHT; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DRUG CONJUGATION; DRUG DELIVERY SYSTEM; DRUG PENETRATION; DRUG TARGETING; EXON; EXON SKIPPING; HUMAN; HUMAN CELL; LIFESPAN; MOUSE; MUSCLE CELL; MUSCLE STRENGTH; NEWBORN; NONHUMAN; PRIORITY JOURNAL; RIGHTING REFLEX; SPINAL MUSCULAR ATROPHY; SYSTEMIC THERAPY; ANIMAL; BLOOD; BRAIN; CELL SURVIVAL; CHEMISTRY; CYTOLOGY; DISEASE MODEL; DRUG EFFECTS; FIBROBLAST; KIDNEY; LIVER CELL; METABOLISM; PHENOTYPE; QUADRICEPS FEMORIS MUSCLE; SYNTHESIS;

ANIMALS; ANIMALS, NEWBORN; APOLIPOPROTEINS E; BIOMARKERS; BLOOD-BRAIN BARRIER; BRAIN; CELL SURVIVAL; DISEASE MODELS, ANIMAL; EXONS; FIBROBLASTS; HEPATOCYTES; HUMANS; KIDNEY; MICE; MORPHOLINOS; MUSCULAR ATROPHY, SPINAL; NANOCONJUGATES; PEPTIDES; PHENOTYPE; QUADRICEPS MUSCLE; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 85020212217     PISSN: 21593337     EISSN: 21593345     Source Type: Journal    
DOI: 10.1089/nat.2016.0652     Document Type: Article

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