Abstract
Cell therapy is moving into clinical experimentation for a number of genetic diseases, including muscular dystrophy. Cell transplantation offers new hopes for so far incurable diseases but the road to success is still long and difficult, since major obstacles remain to be overcome. These include technical hurdles such as isolation, expansion, genetic correction, and storage of cells, validation and diffusion of the protocols. In addition, serious ethical issues such as patient and donor selection, and, related to these, the increasing costs of these therapies need to be solved before cell therapy may move into a standard therapeutic intervention.
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Notes
- 1.
Cytoskeleton  =  a protein network that controls cell shape and its changes during locomotion or contraction
- 2.
Basal lamina  =  a proteic lamina that ensheets each muscle fiber
- 3.
Dystrophin  =  a cytoskeletal protein, absent in Duchenne muscular dystrophy, that maintains muscle fiber integrity
- 4.
Sarcolemma  =  the plasma membrane of the muscle fiber
- 5.
CD133  =  an antigen present on the surface of certain stem cells
- 6.
Notch  =  a membrane protein regulating cell fate
- 7.
mdx/utrophin null  =  a mouse with very severe muscular dystrophy due to simoultaneous absence of dystrophin and the similar protein utrophin
- 8.
Cyclosporine A  =  an immune suppressive drug
- 9.
GMP  =  Good Manifacturing Practice, a method to produce clinical grade cells, necessary for transplantation in patients
- 10.
Non carrier  =  a sister that does not carry the DMD mutation in one X chromosome and is therefore completely healthy
- 11.
cDNA  =  a fragment of DNA corresponding to the mature messenger RNA and able to produce a complete and functional protein
- 12.
kb  =  kilobase, a measure of the length of nucleic acids
- 13.
Integrating vectors  =  viral vectors (usually RNA based) able to insert into the host cell genome
- 14.
Transposons  =  segments of DNA able to move in the mammalian genome from one site to another
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Cossu, G. (2011). Towards a Cell Therapy for Muscular Dystrophy: Technical and Ethical Issues. In: Hug, K., Hermerén, G. (eds) Translational Stem Cell Research. Stem Cell Biology and Regenerative Medicine. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-60761-959-8_6
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