The Role of the Neurofibromin-Syndecan-Cask Complex in the Regulation of Synlaptic RAS-MAPK Signaling and Denoritic Spine Plasticity

Abstract

Neurofibromatosis type 1 (NF1) is a common dominant genetic disorder characterized by multiple benign and malignant tumors of neural origin and often cognitive deficits in children. How mutations in the NF1 gene lead to severe learning deficits is largely unknown. The protein encoded by NF1 neurofibromin contains a GAP domain known to inhibit Ras-mediated signal transduction. The objective of this proposal is to test the hypothesis that the newly identified NF1-Syndecan2-CASK signaling complex plays an essential role in the regulation of synaptic Ras-MAPK activity and dendritic spine maturation. Using several siRNAs and dominant negative constructs for NF1 GAP activity to specifically knockdown or inhibit NF1 we have obtained compelling evidence showing that NF1 deficiency indeed leads to abnormal development of dendritic spines and hyperactive Ras-MAPK activity and furthermore these deficits can be rescued by overexpression of NF1 GRD I, a central domain of NF1 responsible for its Ras GAP activity. Our results have shed new lights on the molecular mechanisms that underlie some of the cognitive deficits in NFI patients and should have important implications for developing effective treatments of this devastating disease.

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Document Details

Document Type
Technical Report
Publication Date
Feb 01, 2007
Accession Number
ADA474457

Entities

People

  • Gang-yi Wu

Organizations

  • Baylor College of Medicine

Tags

DTIC Thesaurus Topics

  • Biomedical Research
  • Brain
  • Cancer
  • Cell Physiological Processes
  • Chemistry
  • Deficiencies
  • Diseases And Disorders
  • Genetic Disorders
  • Growth Factors
  • Learning
  • Maturation
  • Medical Personnel
  • Neoplasms
  • Neurofibromatosis
  • Proteins
  • Regulations
  • Tumor Cell Line

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  • Molecular and Cellular Biology

Technology Areas

  • Biotechnology