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Gene Therapy for Rett Syndrome

Examples

This project aims to develop gene therapy treatments for Rett Syndrome through a collaborative effort between patient advocacy groups and researchers.

Description

Rett Syndrome is a rare neurological disorder with limited treatment options. This project focuses on developing gene therapies to address these unmet needs. The Rett Syndrome Foundation works to support patients with Rett Syndrome, providing resources and advocating for research. Dr. Maria Thompson, an expert in gene therapy at Harvard Medical School, leads the research team. 

Objectives

Develop and test gene therapy treatments for Rett Syndrome.
  • Identify target genes
  • Develop gene vectors
  • Conduct preclinical trials

Project scope

  • Gene identification
  • Vector development
  • Preclinical testing
  • Clinical trials

Method

  • Research Methods:CRISPR-Cas9 gene editing
  • Patient Engagement:Regular consultations with patient groups
  • Data Collection & Analysis:Genetic sequencing and data analysis

Metrics

Successful development of gene therapy vectors
  • Number of vectors developed
  • Preclinical trial results

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