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ICIEM 2017: Gene therapy for IEM, with a special focus on the central nervous system – Part 1

Interview with Dr Charles Vendetti

Dr.+Venditti+Part1

Dr Charles Vendetti, Head of Organic Acid Research Section, and Senior Investigator at the National Human Genome Research Institute, National Institutes of Health, provides a comprehension summary of his talk on gene therapy for inborn errors of metabolism, with a focus on central nervous system disorders.


Paediatric Resource Centre

The Paediatric Resource Centre is dedicated to offering you free access to specially selected peer-reviewed articles, editor’s perspectives, expert interviews, and conference news to keep you informed of the latest developments in the diagnosis, management and treatment of patients with paediatric disorders. These include metabolic disorders and lysosomal storage diseases such as Fabry disease, Pompe disease, Gaucher disease and mucopolysaccharidoses, among others. Led by an Editorial Board of internationally recognised experts Associate Professor Maria Fuller and Dr Katrina Dipple, the Paediatric Resource Centre is editorially independent and freely available to healthcare professionals in Australia and New Zealand. This Resource Centre is hosted by the highly regarded Elsevier journal Molecular Genetics and Metabolism.

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Molecular Genetics and Metabolism is a contribution to the understanding of the metabolic basis of disease. The journal publishes articles describing investigations that use the tools of biochemistry and molecular biology for studies of normal and diseased states. In addition to original research articles, occasional minireviews reporting timely advances as well as brief communications and letters to the editor are considered.

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