Balancing personalized medicine and scalability in base editing for phenylketonuria
Abstract
Translating genome editing approaches to clinical applications can be particularly arduous for diseases with highly heterogeneous genotypes, where individual strategies for hundreds of distinct mutations may each require optimization. Phenylketonuria (PKU), with over 1,000 recorded disease-causing variants,1 exemplifies this scalability challenge.
Fields of Study & Subjects
Metabolism and Genetic Disorders
Pharmacogenetics and Drug Metabolism
Genomics and Rare Diseases
Publication details
- Journal / source
- Molecular Therapy — Nucleic Acids
- Publisher
- Elsevier BV
- Volume
- 37
- Issue
- 1
- Pages
- 102824–102824
- Publication date
- 14 January 2026
- Language
- EN