Developing Xenopus Laevis as a Model to Screen Drugs for Fragile X Syndrome

Abstract

Fragile X Syndrome (FXS) is a neurodevelopmental disorder characterized by deficits including impaired sensory processing and increased susceptibility to seizure. FXS is caused by loss of function of Fragile X Mental Retardation Protein (FMRP). Because FMRP is highly conserved across animals, we have sought to determine whether decreasing FMRP expression in Xenopus may help understand consequences of loss of FMRP. We established a highly sensitive quantitative in vivo imaging assay to evaluate molecular genetic strategies to decrease FMRP expression in brain neurons and demonstrated the capacity to rescue the decreased FMRP expression by gene delivery. We characterized an innate visually-guided avoidance behavior in tadpoles and showed that the avoidance behavior shows rapid and long-lasting improvement after brief periods of training. Decreasing FMRP expression does not significantly impair visual avoidance behavior. We developed a second behavioral assay to evaluate the loss of FMRP in which animals are exposed to seizure-inducing drugs. Decreased FMRP expression increases seizure latency, which was partially compensated by gene delivery of an FMRP homolog. These studies demonstrate promise in developing Xenopus as a system to study FXS.

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Document Details

Document Type
Technical Report
Publication Date
Oct 01, 2013
Accession Number
ADA598718

Entities

People

  • Hollis T. Cline

Organizations

  • Scripps Research

Tags

Communities of Interest

  • Biomedical

DTIC Thesaurus Topics

  • Brain
  • Cells
  • Department Of Defense
  • Diseases And Disorders
  • Fragile-X Syndrome
  • Gene Delivery
  • Gene Therapy
  • Genetic Engineering
  • Intellectual Disability
  • Nervous System
  • Neurodevelopmental Disorders
  • Neurosciences
  • Proteins
  • Retardation
  • Therapy
  • Training

Fields of Study

  • Biology

Readers

  • Aquatic Ecology
  • Child and Adolescent Substance Abuse Science in Autism Spectrum Disorders.
  • Molecular Genetics

Technology Areas

  • Biotechnology