Antisense oligonucleotide therapy in a humanized mouse model of

Animals DNA Copy Number Variations Mental Retardation, X-Linked / therapy Methyl-CpG-Binding Protein 2 / genetics Mice Oligonucleotides, Antisense / therapeutic use

Journal

Science translational medicine
ISSN: 1946-6242
Titre abrégé: Sci Transl Med
Pays: United States
ID NLM: 101505086

Informations de publication

Date de publication:
03 03 2021
Historique:
received: 08 10 2019
revised: 26 05 2020
accepted: 01 10 2020
entrez: 4 3 2021
pubmed: 5 3 2021
medline: 13 7 2021
Statut: ppublish

Résumé

Many intellectual disability disorders are due to copy number variations, and, to date, there have been no treatment options tested for this class of diseases.

Identifiants

DOI: 10.1126/scitranslmed.aaz7785 PMID: 33658357 PMC: PMC8976688
pubmed: 33658357
pii: 13/583/eaaz7785
doi: 10.1126/scitranslmed.aaz7785
pmc: PMC8976688
mid: NIHMS1785194
pii:
doi:

Substances chimiques

Mecp2 protein, mouse 0
Methyl-CpG-Binding Protein 2 0
Oligonucleotides, Antisense 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : NICHD NIH HHS
ID : F32 HD100048
Pays : United States
Organisme : NICHD NIH HHS
ID : P50 HD103555
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS057819
Pays : United States
Organisme : NICHD NIH HHS
ID : U54 HD083092
Pays : United States
Organisme : NINDS NIH HHS
ID : R56 NS100738
Pays : United States
Organisme : NIGMS NIH HHS
ID : R01 GM120033
Pays : United States

Informations de copyright

Copyright © 2021 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.

Références

Genome Biol. 2014;15(12):550
pubmed: 25516281
Nat Biotechnol. 2017 Mar;35(3):249-263
pubmed: 28244991
Pediatrics. 2006 Dec;118(6):e1687-95
pubmed: 17088400
Elife. 2014 Jun 26;3:
pubmed: 24970834
Proc Natl Acad Sci U S A. 2013 Nov 19;110(47):E4530-9
pubmed: 24170860
Neuron. 2012 Jun 21;74(6):1031-44
pubmed: 22726834
Nature. 2015 Feb 19;518(7539):409-12
pubmed: 25470045
Nature. 2011 Oct 05;478(7367):123-6
pubmed: 21979052
Annu Rev Pharmacol Toxicol. 2010;50:259-93
pubmed: 20055705
Am J Hum Genet. 2005 Sep;77(3):442-53
pubmed: 16080119
Am J Pathol. 2013 Jul;183(1):195-210
pubmed: 23684790
Eur J Hum Genet. 2009 Apr;17(4):444-53
pubmed: 18985075
Sci Transl Med. 2012 Dec 5;4(163):163ra158
pubmed: 23220634
Nature. 2017 Apr 20;544(7650):362-366
pubmed: 28405024
N Engl J Med. 2018 Feb 15;378(7):625-635
pubmed: 29443664
Science. 2008 May 30;320(5880):1224-9
pubmed: 18511691
Nat Med. 2017 Oct;23(10):1203-1214
pubmed: 28920956
JCI Insight. 2018 Nov 2;3(21):
pubmed: 30385727
Nat Genet. 2012 Jan 08;44(2):206-11
pubmed: 22231481
Am J Med Genet A. 2010 May;152A(5):1079-88
pubmed: 20425814
Genet Med. 2006 Dec;8(12):784-92
pubmed: 17172942
Mol Ther. 2011 Dec;19(12):2178-85
pubmed: 21971427
Hum Mol Genet. 2004 Nov 1;13(21):2679-89
pubmed: 15351775
Nat Neurosci. 2012 Jan 26;15(2):176-7
pubmed: 22281712
Nat Genet. 1999 Oct;23(2):185-8
pubmed: 10508514
Annu Rev Cell Dev Biol. 2011;27:631-52
pubmed: 21721946
Sci Transl Med. 2011 Mar 2;3(72):72ra18
pubmed: 21368223
Nat Rev Neurol. 2017 Jan;13(1):37-51
pubmed: 27934853
Am J Med Genet. 1999 Jul 30;85(3):243-8
pubmed: 10398236
Bioinformatics. 2013 Jan 1;29(1):15-21
pubmed: 23104886
Proc Natl Acad Sci U S A. 2015 Apr 28;112(17):5509-14
pubmed: 25870282
J Clin Invest. 2006 Aug;116(8):2290-6
pubmed: 16878173
Nature. 2010 Nov 11;468(7321):263-9
pubmed: 21068835
Nature. 2012 Aug 2;488(7409):111-5
pubmed: 22859208
Mol Syndromol. 2012 Apr;2(3-5):128-136
pubmed: 22679399
J Neurosci. 2013 Jul 31;33(31):12887-97
pubmed: 23904623
Hum Mol Genet. 2012 Sep 1;21(17):3806-14
pubmed: 22653753
Hum Mol Genet. 2009 Jun 15;18(12):2188-203
pubmed: 19324899
Nat Rev Genet. 2015 May;16(5):261-75
pubmed: 25732612
Nature. 2015 Dec 3;528(7580):123-6
pubmed: 26605526
Lancet Neurol. 2013 May;12(5):435-42
pubmed: 23541756
J Med Genet. 2005 Feb;42(2):e12
pubmed: 15689435

Auteurs

Yingyao Shao (Y)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030, USA.
ORCID: 0000-0001-6833-2034

Yehezkel Sztainberg (Y)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
ORCID: 0000-0001-8647-7341

Qi Wang (Q)

Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.

Sameer S Bajikar (SS)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
ORCID: 0000-0002-8868-881X

Alexander J Trostle (AJ)

Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.

Ying-Wooi Wan (YW)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.

Paymaan Jafar-Nejad (P)

Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

Frank Rigo (F)

Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

Zhandong Liu (Z)

Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
ORCID: 0000-0002-7608-0831

Jianrong Tang (J)

Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
ORCID: 0000-0001-5664-2692

Huda Y Zoghbi (HY)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA. hzoghbi@bcm.edu.
Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030, USA.
Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Howard Hughes Medical Institute, Baylor College of Medicine, Houston, TX 77030, USA.
ORCID: 0000-0002-0700-3349

Articles similaires

Evaluating the efficacy of telesurgery with dual console SSI Mantra Surgical Robotic System: experiment on animal model and clinical trials.

Sudhir Prem Srivastava, Vishwajyoti Pascual Srivastava, Avinesh Singh et al.
1.00
Robotic Surgical Procedures Animals Humans Telemedicine Models, Animal

Odour generalisation and detection dog training.

Lyn Caldicott, Thomas W Pike, Helen E Zulch et al.
1.00
Animals Odorants Dogs Generalization, Psychological Smell

FBXO22 inhibits colitis and colorectal carcinogenesis by regulating the degradation of the S2448-phosphorylated form of mTOR.

Minle Li, Xuan Chen, Pengfei Qu et al.
1.00
Animals TOR Serine-Threonine Kinases Colorectal Neoplasms Colitis Mice

Use of organic material provided by an automatic enrichment device by weaner pigs and its influence on tail lesions.

Philipp Heseker, Jeanette Probst, Stefanie Ammer et al.
1.00
Animals Tail Swine Behavior, Animal Animal Husbandry

Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Antisense oligonucleotide therapy in a...
questionsmedicales.fr › Phénomènes génétiques › Variation génétique › Polymorphisme génétique › Variation structurale du génome › Variations de nombre de copies de segment d'ADN › Antisense oligonucleotide therapy in a...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Maladies neurodégénératives héréditaires › Retard mental lié à l'X › Antisense oligonucleotide therapy in a...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Nucléoprotéines › Protéines chromosomiques nonhistones › Protéine-2 de liaison au CpG méthylé › Antisense oligonucleotide therapy in a...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Antisense oligonucleotide therapy in a...
questionsmedicales.fr › Actions chimiques et utilisations › Utilisations spécialisées de produits chimiques › Produits chimiques de laboratoire › Sondes moléculaires › Sondes d'acide nucléique › Éléments antisens (génétique) › Oligonucléotides antisens › Antisense oligonucleotide therapy in a...