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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About
Author
Damon Keebler
CRISPR
Gene replacement
Basic science
Started Mon Jan 01 2024
Deadline Wed Dec 31 2025