The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMD
Abstract
:1. Introduction
2. Materials and Methods
2.1. Ethic Statement
2.2. Population Characterization
2.3. Modified Clinical Severity Scale (CSS)
2.4. Cell Culture
2.5. DNA/RNA Purification and Analysis
2.6. Adeno-Associated Virus Vector (AAV) Production and lncRNA Overexpression Experiments (OE)
2.7. Immunohistochemistry
2.8. Bioinformatic Analysis
3. Results
3.1. BMDΔ45-55 Patients lncRNA Breakpoints Impact in lncRNA Sequences
3.2. lncRNA Expression BMD/DMD Patients’ Skeletal Muscle Biopsies
3.3. Low lncRNA Expression during Proliferation in Dystrophic Human Myoblasts
3.4. Low lncRNA Expression during Differentiation in Human Myoblasts
3.5. lncRNA44s2 Overexpression Impacts Human Primary Myoblasts Differentiation
4. Discussion
Supplementary Materials
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Gargaun, E.; Falcone, S.; Solé, G.; Durigneux, J.; Urtizberea, A.; Cuisset, J.M.; Benkhelifa-Ziyyat, S.; Julien, L.; Boland, A.; Sandron, F.; et al. The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMD. Biomedicines 2021, 9, 219. https://doi.org/10.3390/biomedicines9020219
Gargaun E, Falcone S, Solé G, Durigneux J, Urtizberea A, Cuisset JM, Benkhelifa-Ziyyat S, Julien L, Boland A, Sandron F, et al. The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMD. Biomedicines. 2021; 9(2):219. https://doi.org/10.3390/biomedicines9020219
Chicago/Turabian StyleGargaun, Elena, Sestina Falcone, Guilhem Solé, Julien Durigneux, Andoni Urtizberea, Jean Marie Cuisset, Sofia Benkhelifa-Ziyyat, Laura Julien, Anne Boland, Florian Sandron, and et al. 2021. "The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMD" Biomedicines 9, no. 2: 219. https://doi.org/10.3390/biomedicines9020219
APA StyleGargaun, E., Falcone, S., Solé, G., Durigneux, J., Urtizberea, A., Cuisset, J. M., Benkhelifa-Ziyyat, S., Julien, L., Boland, A., Sandron, F., Meyer, V., Deleuze, J. F., Salgado, D., Desvignes, J.-P., Béroud, C., Chessel, A., Blesius, A., Krahn, M., Levy, N., ... Pietri-Rouxel, F. (2021). The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMD. Biomedicines, 9(2), 219. https://doi.org/10.3390/biomedicines9020219