AAV gene therapy for Batten disease

Posted by & filed under Part 16: LYSOSOMAL DISORDERS.

In this work, Beverly Davidson’s group investigated delivery of the gene encoding the soluble lysosomal enzyme tripeptidyl peptidase 1 (TPP1) enzyme to the ependymal cells in a dog model of a Batten disease.  They show that this strategy delayed disease onset, extended life span, and protected dogs from early cognitive decline, suggesting that this approach could improve the lives of children suffering from the same or similar diseases.

http://stm.sciencemag.org/content/7/313/313ra18

Posted by Nicola Brunetti-Pierri

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