[Jacob-list] "Tay-Sachs" disease in Jacob Sheep successfully treated

[Frederick Horak]

TSD is a neurological disease and heretofore seemed insulated from treatment because of the blood brain barrier.  At the 5/15/2013 meeting of the American Society of Gene and Cell Therapy Dr. Douglas Martin (Auburn) formally reported the successful treatment of Tay-Sachs affected Jacobs, the first successful treatment of this disease and by gene therapy.   You can find the complete report at the web site for the ASGCT meeting.  Regards - Fred Horak
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