Mirella Meregalli1, Andrea Farini, Yvan Torrente. 1. Stem Cell Laboratory, Department of Neurological Sciences, University of Milan, Padiglione Ponti, Fondazione IRCCS, Ospedale Policlinico, via Francesco Sforza 35, 20122 Milan, Italy.
Abstract
BACKGROUND: Muscular dystrophies are characterized by primary wasting of skeletal muscle. Mutations in the dystrophin gene cause hereditary muscular diseases such as Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), the most severe form. Characterization of the dystrophin gene and evidence that different types of adult stem cells are capable of muscle regeneration has lead to the development of potential gene therapy and stem cell treatments for DMD. OBJECTIVES: The main goal is to combine gene modification strategies with cell-mediated therapies. This approach could permit autologous transplantation of cells, minimizing the risk of implant rejection. RESULTS/ CONCLUSION: The combination of gene and stem cell approaches seems to be most promising, particularly intra-arterial injections of the patient's own stem cells transduced by antisense oligonucleotide technology. This approach should offer the chance to distribute the autologous corrected stem cells to the whole body musculature providing a clinical benefit for dystrophic patients.
BACKGROUND:Muscular dystrophies are characterized by primary wasting of skeletal muscle. Mutations in the dystrophin gene cause hereditary muscular diseases such as Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), the most severe form. Characterization of the dystrophin gene and evidence that different types of adult stem cells are capable of muscle regeneration has lead to the development of potential gene therapy and stem cell treatments for DMD. OBJECTIVES: The main goal is to combine gene modification strategies with cell-mediated therapies. This approach could permit autologous transplantation of cells, minimizing the risk of implant rejection. RESULTS/ CONCLUSION: The combination of gene and stem cell approaches seems to be most promising, particularly intra-arterial injections of the patient's own stem cells transduced by antisense oligonucleotide technology. This approach should offer the chance to distribute the autologous corrected stem cells to the whole body musculature providing a clinical benefit for dystrophicpatients.
Authors: Ipek Suntar; Antoni Sureda; Tarun Belwal; Ana Sanches Silva; Rosa Anna Vacca; Devesh Tewari; Eduardo Sobarzo-Sánchez; Seyed Fazel Nabavi; Samira Shirooie; Ahmad Reza Dehpour; Suowen Xu; Bahman Yousefi; Maryam Majidinia; Maria Daglia; Giuseppe D'Antona; Seyed Mohammad Nabavi Journal: Acta Pharm Sin B Date: 2020-01-08 Impact factor: 11.413