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