The neurological features of neuronopathic Gaucher disease (nGD), like other LSD’s that affect the CNS, do not respond to ERT. In this paper, Peng et al treated a mouse model with intravenous infusions of iPSC- derived neural stem cells. The preparation used is able to cross the blood brain barrier. There were several beneficial effects of the treatment. Importantly, the levels of acid beta-glucosidase in the brain were higher post treatment and there was some neurological improvement. The authors point out that further work is needed before clinical trials are possible. There are also important challenges of iPSC therapy that need to be overcome. However, an important consideration is that, since no viruses were used, so this approach does not have the disadvantages of gene therapy using viral vectors.
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