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PLoS One 7, e38610, doi:10.1371/ journal.pone.0038610 (2012). 16. Marangolo, P. et al. Improving language without words: first evidence from aphasia. Neuropsychologia 48, 3824–3833, doi:10.1016/ j.neuropsychologia.2010.09.025 (2010). Acknowledgments The study was supported by National ...
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Molecular Therapy Nucleic Acids is an international, open-access journal publishing top-quality basic, translational, and clinical research in the broad fields of nucleic-acid-based therapeutics to treat and/or correct genetic and acquired disease. As an official journal of the American Society of …...
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Replacement therapy is probably most straightforward method of treating monogenic disorders. Once in the cell, the exogenous copy of the correct gene becomes the template for the synthesis of the correct protein, resulting in disease attenuation. Unfortunately, for some monogenic diseases, GT may not...
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