Mechanisms of Chimeric Cell Therapy in Duchenne Muscular Dystrophy
Abstract
:1. Introduction
2. Materials and Methods
2.1. Creation of Human DT-DEC01 Product by PEG-Mediated Fusion of Myoblasts Derived from Normal, Healthy Donor and DMD Patient
2.2. Assessment of Donor-Specific Chimerism by STR-PCR Analysis of Donor Specific STR Loci
2.3. Characterization of the Human DT-DEC01 Product In Vitro by Immunofluorescence Detection of Desmin, Dystrophin and Myosin Heavy Chain
2.4. Assessment of the Intercellular Mitochondrial Transfer and Mitochondrial Chimeric State in the Created DT-DEC01 Product
2.5. Characterization of the Human DT-DEC01 Product In Vitro by Detection of Myotube Formation
3. Results
3.1. Confirmation of Creation of Patient-Derived Chimeric (MBN/MBDMD) DT-DEC01 Product by PEG-Mediated Fusion of Myoblasts from Normal, Healthy Donor and DMD Patient
3.2. Confirmation of Donor-Specific Chimerism in Patient-Derived DT-DEC01 Product through Detection of the STR Loci Specific for the Normal Myoblast Donor
3.3. Confirmation of Myogenic Potential of the Patient-Derived DT-DEC01 Product through Expression of Desmin, Dystrophin and Myosin Heavy Chain
3.4. Confirmation of Transfer of Healthy Donor Mitochondria and Presence of Chimeric Mitochondria in the Patients-Derived DT-DEC01 Product
3.5. Confirmation of Myogenic Potential of the Patient-Derived DT-DEC01 Product by Detection of Myotube Formation
4. Discussion
5. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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ID of the MBN/MBDMD DT-DEC01 Product | Donor (MBN)-Specific STR Loci |
---|---|
MBN1/MBDMD1 | 27% |
MBN2/MBDMD2 | 21% |
MBN3/MBDMD3 | 6% |
MBN4/MBDMD4 | 35% |
MBN5/MBDMD5 | 15% |
MBN6/MBDMD6 | 6% |
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Siemionow, M.; Ziemiecka, A.; Bożyk, K.; Siemionow, K. Mechanisms of Chimeric Cell Therapy in Duchenne Muscular Dystrophy. Biomedicines 2024, 12, 1996. https://doi.org/10.3390/biomedicines12091996
Siemionow M, Ziemiecka A, Bożyk K, Siemionow K. Mechanisms of Chimeric Cell Therapy in Duchenne Muscular Dystrophy. Biomedicines. 2024; 12(9):1996. https://doi.org/10.3390/biomedicines12091996
Chicago/Turabian StyleSiemionow, Maria, Anna Ziemiecka, Katarzyna Bożyk, and Krzysztof Siemionow. 2024. "Mechanisms of Chimeric Cell Therapy in Duchenne Muscular Dystrophy" Biomedicines 12, no. 9: 1996. https://doi.org/10.3390/biomedicines12091996