Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

Journal of Clinical Investigation

Volumn 124, Issue 9, 2014, Pages 4067-4081

  Bestas, Burcu ^a   Moreno, Pedro M D ^a,b   Blomberg, K Emelie M ^a   Mohammad, Dara K ^a   Saleh, Amer F ^c,j   Sutlu, Tolga ^a,k   Nordin, Joel Z ^a   Guterstam, Peter ^d   Gustafsson, Manuela O ^a   Kharazi, Shabnam ^a   Piątosa, Barbara ^e   Roberts, Thomas C ^f,g   Behlke, Mark A ^h   Wood, Matthew J A ^f   Gait, Michael J ^c   Lundin, Karin E ^a   El Andaloussi, Samir ^a,f   Mansson, Robert ^a   Berglöf, Anna ^a   Wengel, Jesper ⁱ   Smith, C I Edvard ^a   more..

^a KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

^b UNIVERSITY OF PORTO   (Portugal)

^c MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

^d STOCKHOLM UNIVERSITY   (Sweden)

^e CHILDREN'S MEMORIAL HEALTH INSTITUTE   (Poland)

^f UNIVERSITY OF OXFORD   (United Kingdom)

^g SCRIPPS RESEARCH INSTITUTE   (United States)

^h INTEGRATED DNA TECHNOLOGIES   (United States)

ⁱ UNIVERSITY OF SOUTHERN DENMARK   (Denmark)

^j ASTRAZENECA   (United Kingdom)

^k SABANCI UNIVERSITY   (Turkey)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; B LYMPHOCYTE RECEPTOR; BRUTON TYROSINE KINASE; MESSENGER RNA; SPLICE CORRECTING OLIGONUCLEOTIDE; UNCLASSIFIED DRUG; AGAMMAGLOBULINAEMIA TYROSINE KINASE; LUCIFERASE; OLIGONUCLEOTIDE; PROTEIN TYROSINE KINASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTISENSE THERAPY; ARTICLE; B LYMPHOCYTE; CELL SURVIVAL; CONTROLLED STUDY; DRUG DESIGN; DRUG STRUCTURE; ENZYME ACTIVATION; GENE EXPRESSION REGULATION; GENE MUTATION; GENE TARGETING; HUMAN; HUMAN CELL; KNOCKOUT MOUSE; MOUSE; NONHUMAN; PRE B LYMPHOCYTE; PROTEIN EXPRESSION; TRANSGENIC MOUSE; X LINKED AGAMMAGLOBULINEMIA; AGAMMAGLOBULINEMIA; ANIMAL; CELL CULTURE; ENZYMOLOGY; GENETIC DISEASES, X-LINKED; GENETICS; METABOLISM; MONOCYTE; PHYSIOLOGY; RNA SPLICING;

AGAMMAGLOBULINEMIA; ANIMALS; B-LYMPHOCYTES; CELLS, CULTURED; GENETIC DISEASES, X-LINKED; HUMANS; LUCIFERASES; MICE, TRANSGENIC; MONOCYTES; OLIGONUCLEOTIDES; PROTEIN-TYROSINE KINASES; RNA SPLICING;

EID: 84907008349     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI76175     Document Type: Article

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