Functional analysis of splicing mutations in the IDS gene and the use of antisense oligonucleotides to exploit an alternative therapy for MPS II

Biochimica et Biophysica Acta - Molecular Basis of Disease

Volumn 1852, Issue 12, 2015, Pages 2712-2721

  Matos, Liliana ^a,b   Gonçalves, Vânia ^c   Pinto, Eugénia ^d   Laranjeira, Francisco ^d   Prata, Maria João ^b,e   Jordan, Peter ^c   Desviat, Lourdes R ^f,g,h   Pérez, Belén ^f,g,h   Alves, Sandra ^a  

^a NATIONAL INSTITUTE OF HEALTH   (Portugal)

^b UNIVERSITY OF PORTO   (Portugal)

^c NATIONAL INSTITUTE OF HEALTH DR RICARDO JORGE   (Portugal)

^d UP   (Portugal)

^e UNIVERSITY OF PORTO   (Portugal)

^f UNIVERSIDAD AUTÓNOMA DE MADRID   (Spain)

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

^h HOSPITAL UNIVERSITARIO LA PAZ IDIPAZ   (Spain)

Author keywords

Antisense therapy; IDS gene; Lysosomal storage disorders; Splicing regulation

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CIS ACTING ELEMENT; HETEROGENEOUS NUCLEAR RIBONUCLEOPROTEIN;

ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; GENE MUTATION; GENE REPRESSION; GENETIC TRANSFECTION; GENOTYPE; HUMAN; HUMAN CELL; HUNTER SYNDROME; IDS GENE; MALE; MARKER GENE; PRIORITY JOURNAL; RNA SPLICING;

EID: 84943645882     PISSN: 09254439     EISSN: 1879260X     Source Type: Journal    
DOI: 10.1016/j.bbadis.2015.09.011     Document Type: Article

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