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While there is a commercially available treatment for the systemic manifestations, neuropathology still remains untreatable. We previously demonstrated that gene therapy represents a feasible therapeutic tool for the treatment of the neuronopathic forms of Gaucher disease (nGD). In order to further enhance the therapeutic affects to the central nervous system, we systemically delivered an adeno-associated virus (AAV) serotype 9 carrying the human GBA gene under control of a neuron-specific promoter to an nGD mouse model. Gene therapy increased the life span of treated animals, rescued the lethal neurodegeneration, normalized the locomotor behavioural defects and ameliorated the visceral pathology. Together, these results provided further indication of gene therapy as a possible effective treatment option for the neuropathic forms of Gaucher disease.<\/jats:p>","DOI":"10.1093\/hmg\/ddz317","type":"journal-article","created":{"date-parts":[[2019,12,20]],"date-time":"2019-12-20T15:11:22Z","timestamp":1576854682000},"page":"1933-1949","source":"Crossref","is-referenced-by-count":42,"title":["Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes"],"prefix":"10.1093","volume":"29","author":[{"given":"Giulia","family":"Massaro","sequence":"first","affiliation":[{"name":"UCL School of Pharmacy, University College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael P","family":"Hughes","sequence":"additional","affiliation":[{"name":"UCL School of Pharmacy, University College London, London, 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