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2 Skin and Musculoskeletal Tissues
Pages 9-18

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From page 9...
... Autologous and allogeneic skin therapies have a long history, from skin grafts that were performed more than 4,000 years ago to modernday hair transplants, said Anthony Oro, a professor of dermatology at Stanford University. Remarkable advances have been made in the ability to grow healthy skin and musculoskeletal tissues for potential use in the 9
From page 10...
... He and his team have been working on the regeneration of autologous skin tissue in order to treat DEB. In 2014 Oro's team conducted Phase I clinical trials on an autologous retroviralcorrected keratinocyte sheet product called LEAES (for LZRSE-Col7A1 engineered autologous epidermal sheet)
From page 11...
... In order to address these issues, Oro and his collaborators used a new process called therapeutic reprogramming, in which iPS cells are created from the patient's skin cells and genome editing is used to correct the mutation that causes DEB. The edited cells are screened to select for those that do not have mutations for squamous cell carcinoma, and then downstream differentiation techniques are used to make human skin cells for transplantation (Sebastiano et al., 2014)
From page 12...
... . o e TRANSLAT T TION OF RE ESEARCH D ISCOVERIE INTO ES CLIN NICAL CARE RE Th here are nume erous opportu unities for the use of cell-b e based therapi ies in the treatment of skin and mu f usculoskeletal tissues.
From page 13...
... . TABLE 2-1 Challenges and Opportunities to Profitability of Cell-Based Therapies Challenge Opportunity Cost Increase efficiencies, standards Time Streamline studies required Technical difficulty Identify standards for cell sourcing Clinical uncertainty Improved clinical databases Regulatory uncertainty Develop standards Manufacturing Improve scalability, standards Predicted market opportunity Increase availability of data International opportunities Improve harmonization of standards SOURCE: Anthony Ratcliffe, National Academies of Sciences, Engineering, and Medicine workshop presentation, October 13, 2016.
From page 14...
... The organization's mission is to accelerate DMD research, advocate for DMD causes, demand optimal care for all young men with DMD, educate the global community, and, ultimately, end DMD. PPMD was instrumental in passing the Muscular Dystrophy Care Act in 2001,3 which has delivered $700 million into muscular dystrophy research and development.
From page 15...
... Early studies in mice by Capricor have demonstrated that the delivery of cardiosphere exosomes also results in the growth of new heart cells. However, mouse models may be of minimal value, Furlong said, commenting that "the mouse has been treated and cured many, many times; the boys not very many times." Furlong described several other avenues that are being pursued, including the use of iPS cells to create more effective disease models to study cardiomyopathy and skeletal muscle pathologies.
From page 16...
... She argued that in a "rare disease with a high unmet medical need and no options," therapies that are safe and potentially beneficial need to be "out there on the market" so that patients can use them and researchers can learn more about how and whether the therapies work. One insurance company has already announced that it will not pay for the Sarepta therapy, indicating that even therapies with regulatory approval may encounter challenges in entering the market.6 This potential roadblock could prevent companies or investors from initially investing in therapies that are not guaranteed commercial success.
From page 17...
... The lack of a perfect animal model means that tough decisions will have to be made about how much evidence is needed before moving a potential therapy into humans, Furlong said. Oro concurred, noting that mouse and human skin are quite different, so clinical trials must move forward with only partial results from the animal model.
From page 18...
... The topic of scaling up and expanding cell populations for use in cellular therapies was brought up by a workshop participant. How can scientists and manufacturers make sure that their expanded cell populations retain purity and potency?


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