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| Full Title | Pioneering Stem Cell/Gene Therapy Trials 1st Edition |
|---|---|
| Author(s) | Roger Bertolotti, Keiyo Ozawa, H. Kirk Hammond |
| Edition | 1st Edition |
| ISBN | 9781040189764, 9789067643948, 9781482298482, 9780429076886 |
| Publisher | CRC Press |
| Format | PDF and EPUB |
The first unequivocal success for Gene Therapy was reported in April 2000 for X-SCID patients. Pioneering stem cell/gene therapy clinical trials are the focus of this book. Therapy successes such as the X-SCID trial and improved ADA-SCID ones are presented together with pioneering angio/vasculogenic clinical trials mediated either by transient gene therapy or emerging autologous stem cell transplantation. Highlights also include 1) promises of the breakthrough combination of stem cell- and transient gene-therapy, 2) gene therapy trials for neurodegenerative disease on non-human primates where long-term gene therapy might involve brain stem cells, and 3) the first clinical trial with non-invasive monitoring of therapeutic gene expression as a prospective conclusion.
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