A Coordinated Attack: Rett Syndrome Therapeutic Development.

Animals Drug Discovery / methods Drug Evaluation, Preclinical / methods Drug Repositioning Female Humans Methyl-CpG-Binding Protein 2 / genetics Mutation Research Design Rett Syndrome / drug therapy
Rett syndrome pharmacological intervention

Journal

Trends in pharmacological sciences
ISSN: 1873-3735
Titre abrégé: Trends Pharmacol Sci
Pays: England
ID NLM: 7906158

Informations de publication

Date de publication:
04 2019
Historique:
received: 16 11 2018
revised: 06 02 2019
accepted: 06 02 2019
entrez: 26 3 2019
pubmed: 25 3 2019
medline: 17 4 2019
Statut: ppublish

Résumé

Rett syndrome (RTT) is a neurodevelopmental disorder caused by mutations in the Methyl CpG binding protein 2 (MeCP2) gene. This Science & Society article focuses on pharmacological strategies that attack RTT treatment from multiple angles, including drug repurposing and de novo discovery efforts, and discusses the impacts of preclinical study design and translationally relevant outcome measures.

Identifiants

DOI: 10.1016/j.tips.2019.02.007 PMID: 30905360 PMC: PMC6482467
pubmed: 30905360
pii: S0165-6147(19)30038-0
doi: 10.1016/j.tips.2019.02.007
pmc: PMC6482467
mid: NIHMS1024467
pii:
doi:

Substances chimiques

MECP2 protein, human 0
Methyl-CpG-Binding Protein 2 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

233-236

Subventions

Organisme : NIMH NIH HHS
ID : K01 MH112983
Pays : United States
Organisme : NIMH NIH HHS
ID : R01 MH113543
Pays : United States

Informations de copyright

Copyright © 2019 Elsevier Ltd. All rights reserved.

Références

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Auteurs

Rocco G Gogliotti (RG)

Vanderbilt Department of Pharmacology and Vanderbilt Center for Neuroscience Drug Discovery, Vanderbilt University, Nashville, TN, USA.

Colleen M Niswender (CM)

Vanderbilt Department of Pharmacology and Vanderbilt Center for Neuroscience Drug Discovery, Vanderbilt University, Nashville, TN, USA; Vanderbilt Institute of Chemical Biology, Vanderbilt University, Nashville, TN, USA; Vanderbilt Kennedy Center, Vanderbilt University Medical Center, Nashville, TN, USA. Electronic address: Colleen.niswender@vanderbilt.edu.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Techniques d'investigation › Développement de médicament › Découverte de médicament › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Techniques d'investigation › Évaluation préclinique de médicament › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Techniques d'investigation › Développement de médicament › Repositionnement des médicaments › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Nucléoprotéines › Protéines chromosomiques nonhistones › Protéine-2 de liaison au CpG méthylé › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Phénomènes génétiques › Variation génétique › Mutation › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Disciplines des sciences naturelles › Science › Recherche › Plan de recherche › A Coordinated Attack: Rett Syndrome...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Maladies neurodégénératives héréditaires › Retard mental lié à l'X › Syndrome de Rett › A Coordinated Attack: Rett Syndrome...