Monogenic Diseases – Caused by Mutations in a Single Gene
Utilizing BlueAllele’s proprietary PALIDON™ repair templates, BlueAllele is designing gene editing therapeutics for patients to fix disease-causing genetic mutations.
Healthy Gene
Normal Protein Expression and Function
Different Types of DNA Mutations
Abnormal Protein Expression
Loss-of-Function Diseases
- Potential BlueAllele targets:
- Hemophilia
- Fabry disease
- Gaucher's disease
- Cystic fibrosis
- Retinitis pigmentosa
Gain-of-Function Diseases
- Potential BlueAllele targets:
- Myotonic dystrophy
- Ataxia
- Huntington disease
- Parkinson's disease
- Alzheimer disease