Seeking stability for gene addition in inborn errors of metabolism
Abstract
So-called inborn errors of metabolism (IOMs), such as phenylketonuria (PKU), have long been treated through dietary therapy consisting of dietary restrictions accompanied by supplementation with specific amino acids, carbohydrates, fatty acids, and/or vitamin cofactors. While dietary therapy has demonstrated benefits, including for PKU, its limitations have led to the development of alternative approaches, including gene therapy. However, marketing approval for gene therapy has been slow, at least relative to the expectations of patients and care providers, and the uptake of approved gene therapies has been disappointing in many cases.
Fields of Study & Subjects
Virus-based gene therapy research
Lysosomal Storage Disorders Research
RNA Interference and Gene Delivery
Publication details
- Journal / source
- Molecular Therapy — Nucleic Acids
- Volume
- 37
- Issue
- 1
- Pages
- 102856–102856
- Publication date
- 16 February 2026
- Language
- EN