Translating the Lessons From Gene Therapy to the Development of Regenerative Medicine
Division of Cancer Biology, Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA.Molecular Therapy (Impact Factor: 6.23). 03/2011; 19(3):439-41. DOI: 10.1038/mt.2011.14
mt is a cross-disciplinary biomedical journal devoted to publishing the most exciting advances in pharmacology and therapeutics, as they pertain to advances in translational and clinical medicine. It is recognized as one of the most prestigious journals in the field. With an impact factor of 6.825*, mt ranks in the top 4.2% of scientific journals in the latest Science Citation Index. Published monthly online and in print.
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Article: Translating the Lessons From Gene Therapy to the Development of Regenerative Medicine
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ABSTRACT: INTRODUCTION: Lysosomal storage disorders (LSDs) encompass more than 50 distinct diseases, caused by defects in various aspects of lysosomal function. Neurodegeneration and/or dysmyelination are the hallmark of roughly 70% of LSDs. Gene therapy represents a promising approach for the treatment of CNS manifestations in LSDs, as it has the potential to provide a permanent source of the deficient enzyme, either by direct injection of vectors or by transplantation of gene-corrected cells. In this latter approach, the biology of neural stem/progenitor cells and hematopoietic cells might be exploited. AREAS COVERED: Based on an extensive literature search up until March 2011, the author reviews and discusses the progress, the crucial aspects and the major challenges towards the development of novel gene therapy strategies aimed to target the CNS, with particular attention to direct intracerebral gene delivery and transplantation of neural stem/progenitor cells. EXPERT OPINION: The implementation of viral vector delivery systems with specific tropism, regulated transgene expression, low immunogenicity and low genotoxic risk and the improvement in isolation and manipulation of relevant cell types to be transplanted, are fundamental challenges to the field. Also, combinatorial strategies might be required to achieve full correction in LSDs with neurological involvement.Expert opinion on biological therapy 05/2011; 11(9):1153-67. DOI:10.1517/14712598.2011.582036 · 3.74 Impact Factor
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