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| Pàgina inicial > Articles > Articles publicats > Platelet Derived Growth Factor-AA Correlates With Muscle Function Tests and Quantitative Muscle Magnetic Resonance in Dystrophinopathies |
(University Hospital Antwerp (Bèlgica)) (Newcastle University) (Hospital Sant Joan de Déu (Manresa)) (Hospital Sant Joan de Déu (Barcelona, Catalunya)) (Institut d'Investigació Biomèdica Sant Pau) (Newcastle University) (Centro de Investigación Biomédica en Red de Enfermedades Raras) (Universitat de Barcelona. Departament de Genètica, Microbiologia i Estadística) (Institut d'Investigació Biomèdica Sant Pau) (Institut d'Investigació Biomèdica Sant Pau) (Philips Healthcare Iberia) (Centro de Investigación Biomédica en Red de Enfermedades Raras) (Centro de Investigación Biomédica en Red de Enfermedades Raras) (Centro de Investigación Biomédica en Red de Enfermedades Raras) (Newcastle University)
| Data: | 2021 |
| Resum: | Introduction: Duchenne (DMD) and Becker (BMD) muscular dystrophy are X-linked muscular disorders produced by mutations in the DMD gene which encodes the protein dystrophin. Both diseases are characterized by progressive involvement of skeletal, cardiac, and respiratory muscles. As new treatment strategies become available, reliable biomarkers and outcome measures that can monitor disease progression are needed for clinical trials. Methods: We collected clinical and functional data and blood samples from 19 DMD patients, 13 BMD patients, and 66 healthy controls (8 pediatric and 58 adult controls), and blood samples from 15 patients with dysferlinopathy (DYSF) and studied the serum concentration of 4 growth factors involved in the process of muscle fibrosis. We correlated the serum concentration of these growth factors with several muscle function tests, spirometry results and fat fraction identified by quantitative Dixon muscle MRI. Results: We found significant differences in the serum concentration of Platelet Derived Growth Factor-AA (PDGF-AA) between DMD patients and pediatric controls, in Connective Tissue Growth Factor (CTGF) between BMD patients and adult controls, and in and Transforming Growth Factor- β1 (TGF-β1) between BMD and DYSF patients. PDGF-AA showed a good correlation with several muscle function tests for both DMD and BMD patients and with thigh fat fraction in BMD patients. Moreover, PDGF-AA levels were increased in muscle biopsies of patients with DMD and BMD as was demonstrated by immunohistochemistry and Real-Time PCR studies. Conclusion: Our study suggests that PDGF-AA should be further investigated in a larger cohort of DMD and BMD patients because it might be a good biomarker candidate to monitor the progression of these diseases. |
| Ajuts: | Instituto de Salud Carlos III PI15/01822 Instituto de Salud Carlos III PI18/1525 Instituto de Salud Carlos III PI19/00122 Instituto de Salud Carlos III CD18/00195 |
| Drets: | Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.  |
| Llengua: | Anglès |
| Document: | Article ; recerca ; Versió publicada |
| Matèria: | PDGF ; MRI ; Biomarker ; Duchenne ; Becker ; Dystrophinopathy |
| Publicat a: | Frontiers in neurology, Vol. 12 (june 2021) , ISSN 1664-2295 |
