A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold. The implications of these promising results for developing similar gene therapies for use in hu-mans and for targeting additional brain disorders are discussed in two articles published in Human Gene Therapy.
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Promising results with new gene therapy approach for treating inherited neurodegenerative diseases
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