Discovery of Novel Therapeutics for Disordered Sleep in Fragile X Syndrome

Abstract

Here we employed a large-scale screen of FDA approved/clinical compounds to identify compounds that can modify disrupted sleep or circadian rhythms deficits in a validated fruit fly (Drosophila) model of Fragile X. We identify and validate a high value compound penfluridol that can address a subset of circadian and sleep phenotypes. Penfluridol is a dopamine D2 receptor and T-type calcium channel blocker highlighting a potential role for these molecular targets for therapeutic strategies. The identification of a potential therapeutic for Fragile X is a major step forward in the development of newtreatments for this neurodevelopmental disorder.

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Document Details

Document Type
Technical Report
Publication Date
Jun 01, 2021
Accession Number
AD1151201

Entities

People

  • Ravi Allada

Organizations

  • Northwestern University

Tags

DTIC Thesaurus Topics

  • Animals
  • Autism
  • Biological Therapy
  • Biomedical Research
  • Circadian Rhythms
  • Diptera
  • Diseases And Disorders
  • Drosophila
  • Fragile-X Syndrome
  • Genes
  • Genetics
  • Maryland
  • Medical Personnel
  • Neurodevelopmental Disorders
  • Phenotypes
  • Professional Development
  • Spectra
  • Standards
  • Students
  • Technology Transfer
  • Therapy
  • Toxicity

Fields of Study

  • Biology

Readers

  • Circadian Sleep-Wake Regulation and Chronobiology
  • Marine Ecological Systems Migration
  • Neuroscience