New Targeted Treatments for Fragile X Syndrome.
Animals
Cellular Reprogramming
/ genetics
Child
DNA Methylation
Disease Models, Animal
Epigenesis, Genetic
Fragile X Mental Retardation Protein
Fragile X Syndrome
/ drug therapy
GABA Agonists
/ pharmacology
Gene Expression Regulation
/ drug effects
Gene Silencing
/ drug effects
Humans
Molecular Targeted Therapy
/ trends
Selective Serotonin Reuptake Inhibitors
/ pharmacology
Signal Transduction
Treatment Outcome
Fragile X syndrome
acamprosate
cannabidiol
lovastatin
metformin
minocycline
sertraline
targeted treatment.
Journal
Current pediatric reviews
ISSN: 1875-6336
Titre abrégé: Curr Pediatr Rev
Pays: United Arab Emirates
ID NLM: 101240290
Informations de publication
Date de publication:
2019
2019
Historique:
received:
24
01
2019
revised:
14
05
2019
accepted:
21
05
2019
pubmed:
27
6
2019
medline:
24
6
2020
entrez:
27
6
2019
Statut:
ppublish
Résumé
Fragile X Syndrome (FXS) is the most common cause of inherited intellectual disability with prevalence rates estimated to be 1:5,000 in males and 1:8,000 in females. The increase of >200 Cytosine Guanine Guanine (CGG) repeats in the 5' untranslated region of the Fragile X Mental Retardation 1 (FMR1) gene results in transcriptional silencing on the FMR1 gene with a subsequent reduction or absence of fragile X mental retardation protein (FMRP), an RNA binding protein involved in the maturation and elimination of synapses. In addition to intellectual disability, common features of FXS are behavioral problems, autism, language deficits and atypical physical features. There are still no currently approved curative therapies for FXS, and clinical management continues to focus on symptomatic treatment of comorbid behaviors and psychiatric problems. Here we discuss several treatments that target the neurobiological pathway abnormal in FXS. These medications are clinically available at present and the data suggest that these medications can be helpful for those with FXS.
Identifiants
pubmed: 31241016
pii: CPR-EPUB-99190
doi: 10.2174/1573396315666190625110748
pmc: PMC6930353
mid: NIHMS1044645
doi:
Substances chimiques
FMR1 protein, human
0
GABA Agonists
0
Serotonin Uptake Inhibitors
0
Fragile X Mental Retardation Protein
139135-51-6
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
251-258Subventions
Organisme : NICHD NIH HHS
ID : U54 HD079125
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR001860
Pays : United States
Informations de copyright
Copyright© Bentham Science Publishers; For any queries, please email at epub@benthamscience.net.
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