Assessing Gene Therapy Efficacy in Infantile-Onset Pompe Disease
We report the novel finding of a widespread reduction in myocardial native T1 values and size of abnormal regions following gene therapy in a pediatric patient with infantile-onset Pompe disease. We emphasize the importance of serial cardiac magnetic resonance in assessing the efficacy of gene therapy and monitoring myocardial alterations in Pompe disease.
Authors
- Jinhao Lyu (ORCID: https://orcid.org/0000-0002-5177-7640)
- Xin Lou (ORCID: https://orcid.org/0000-0002-6715-0375)
- Wanbing Wang
- Fei Yan (ORCID: https://orcid.org/0000-0002-5870-2799)
- Xinghua Zhang (ORCID: https://orcid.org/0000-0002-9487-191X)
Institutions
- Chinese PLA General Hospital (CN)
Publication Details
- Journal
- JACC Case Reports
- Published
- 2024-08-01
- DOI
- https://doi.org/10.1016/j.jaccas.2024.102446
- Citations
- 2
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.65