Challenges and opportunities to bridge translational to clinical research for personalized mitochondrial medicine
Document Type
Journal Article
Publication Date
1-19-2024
Journal
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
Volume
21
Issue
1
DOI
10.1016/j.neurot.2023.e00311
Keywords
Clinical trial; Energy metabolism; Mitochondrial medicine; Next generation therapeutics; Patient-centric approach
Abstract
Mitochondrial disorders are a group of rare and heterogeneous genetic diseases characterized by dysfunctional mitochondria leading to deficient adenosine triphosphate synthesis and chronic energy deficit in patients. The majority of these patients exhibit a wide range of phenotypic manifestations targeting several organ systems, making their clinical diagnosis and management challenging. Bridging translational to clinical research is crucial for improving the early diagnosis and prognosis of these intractable mitochondrial disorders and for discovering novel therapeutic drug candidates and modalities. This review provides the current state of clinical testing in mitochondrial disorders, discusses the challenges and opportunities for converting basic discoveries into clinical settings, explores the most suited patient-centric approaches to harness the extraordinary heterogeneity among patients affected by the same primary mitochondrial disorder, and describes the current outlook of clinical trials.
APA Citation
Gropman, Andrea L.; Uittenbogaard, Martine N.; and Chiaramello, Anne E., "Challenges and opportunities to bridge translational to clinical research for personalized mitochondrial medicine" (2024). GW Authored Works. Paper 4149.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/4149
Department
Anatomy and Regenerative Biology