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Genetics and Therapies for GM2 Gangliosidosis NEWS RELEASE: 27-04-2018

The article by Maria Begona Cachon-Gonzalez and colleagues is published in Current Gene Therapy, Volume 18, Issue 2, 2018

Cachón-González MB, Zaccariotto E, Cox TM. Genetics and Therapies for GM2 Gangliosidosis, Curr Gene Ther. 2018, Vol 18. DOI: 10.2174/1566523218666180404162622


Cachón-González MB, Wang SZ, Lynch A, et al. Effective gene therapy in an authentic model of Tay-Sachs-related diseases. Proc Natl Acad Sci USA 2006; 103(27): 10373-8

Bradbury AM, Cochran JN, McCurdy VJ, et al. Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy. Mol Ther 2013; 21(7): 1306-15

Gray-Edwards HL, Randle AN, Maitland SA et al. Adeno-Associated Virus gene therapy in a sheep model of Tay-Sachs disease. Hum Gene Ther 2017; 29: 312-326

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