Present and future of antisense therapy for splicing modulation in inherited metabolic disease

Journal of Inherited Metabolic Disease

Volumn 33, Issue 4, 2010, Pages 397-403

  Pérez, Belen ^a,b   Rodríguez Pascau, Laura ^b,c   Vilageliu, Luisa ^b,c   Grinberg, Daniel ^b,c   Ugarte, Magdalena ^a,b   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

ANTISENSE OLIGONUCLEOTIDE; MESSENGER RNA; COMPLEMENTARY DNA;

ANTISENSE THERAPY; DISEASE MODEL; DNA BINDING; DRUG DELIVERY SYSTEM; DUCHENNE MUSCULAR DYSTROPHY; FEASIBILITY STUDY; GENE MUTATION; GENE TARGETING; GENETIC CODE; GENETIC TRANSCRIPTION; HUMAN; INBORN ERROR OF METABOLISM; INTRON; MOLECULAR RECOGNITION; MOLECULAR STABILITY; NONHUMAN; PROTEIN PROCESSING; REVIEW; ALTERNATIVE RNA SPLICING; GENE THERAPY; GENETICS; METABOLIC DISEASES; PROCEDURES; TRENDS;

ALTERNATIVE SPLICING; DNA, ANTISENSE; GENE THERAPY; HUMANS; METABOLIC DISEASES; GENETIC THERAPY;

EID: 77956060846     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-010-9135-1     Document Type: Review

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