Leveraging Transgenic Animal Models for Robust In Vivo Validation of Therapeutic Targets in Inherited Retinal Diseases
Time: 4:00 pm
day: Conference Day One
Details:
- Choosing disease-specific knockout mouse models to accurately mirror human genetic pathology and provide relevant efficacy data
- Collaborating with pre-clinical partners experienced in retinal gene delivery to optimize vector administration protocols
- Collecting longitudinal functional and histological evidence of therapeutic benefit to build compelling proof-of-concept data supporting regulatory confidence
