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Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS

Injection of AAV–shRNA below the pial surface of the spinal cord prevents onset or ameliorates progression in a mouse model of ALS, and achieves widespread delivery to the spinal cord and brain motor centers in adult pigs and non-human primates.

Mariana Bravo-Hernandez
Takahiro Tadokoro
Martin Marsala
Research23 Dec 2019
Nature Medicine

Volume: 26, P: 118-130

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Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS

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