Towards a Cell Therapy for Muscular Dystrophy: Technical and Ethical Issues
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.
KeywordsCell therapy Muscular dystrophy Patient selection Rare diseases Trial costs
- 36.Hyun I, Lindvall O, Ahrlund-Richter L, Cattaneo E, Cavazzana-Calvo M, Cossu G et al. New ISSCR guidelines underscore major principles for responsible translational stem cell research. Cell Stem Cell 2008; 4:607–9.Google Scholar