Antisense mediated splicing modulation for inherited metabolic diseases: Challenges for delivery

Nucleic Acid Therapeutics

Volumn 24, Issue 1, 2014, Pages 48-56

  Pérez, Belen ^a,b   Vilageliu, Lluisa ^c   Grinberg, Daniel ^c   Desviat, Lourdes R ^a,b  

^a UNIVERSIDAD AUTÓNOMA DE MADRID   (Spain)

^b CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED DE ENFERMEDADES RARAS CIBERER   (Spain)

^c UNIVERSITY OF BARCELONA   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

AMINO ACID DERIVATIVE; ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B100; MIPOMERSEN; PHENYLALANINE 4 MONOOXYGENASE; UREA;

ANTISENSE THERAPY; CENTRAL NERVOUS SYSTEM; CLINICAL TRIAL (TOPIC); ENZYME DEFECT; HUMAN; INHERITANCE; INHERITED METABOLIC DISEASE; LIVER; METABOLIC DISORDER; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; REVIEW; RNA SPLICING; TARGET ORGAN;

ANIMALS; CENTRAL NERVOUS SYSTEM; DRUG DELIVERY SYSTEMS; EXONS; GENETIC DISEASES, INBORN; HUMANS; LIVER; MALE; METABOLISM, INBORN ERRORS; MICE; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING; TARGETED GENE REPAIR;

EID: 84893589979     PISSN: 21593337     EISSN: None     Source Type: Journal    
DOI: 10.1089/nat.2013.0453     Document Type: Review

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