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

[Frederick Horak]

Dear Zach Oaster - The work on the sheep model has been successful for two years and they began primate studies six or eight months ago.  One of the monkeys died.  The phase one human trial which was targeted for this Fall depended on the successful primate tests.   The collaborators think they know why the monkey died and tests on primates are being rescheduled for this Fall.  In the meantiome, they are continuing work on the sheep model;  therapies for delivery of the vectore are being refined to avoid surgical intrusion and test an injection method.  

The research on this model is going in several different directions from vector to delivery and delivery of therapies for the more than forty related neural diseases.  The monogenic types, like the Jacob model, are easiest to solve but explosions in the human genome and role of genes unknown just years ago is enabling many breakthroughs.
Regards - Fred Horak

-----Original Message-----
From: Zach Oaster <zach at fattoaster.com>
To: Frederick Horak <jacobflock at aol.com>
Cc: jacob-list <jacob-list at jacobsheep.com>; fhorak <fhorak at aol.com>
Sent: Fri, Jun 7, 2013 2:38 pm
Subject: Re: [Jacob-list] "Tay-Sachs" disease in Jacob Sheep successfully treated


WOW, that is amazing news. Do they have a timeline in terms of how long it might be before they can translate that into a human treatment?



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Zach Oaster
zach at fattoaster.com
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Visit Zach & Lindsay's farm blog: http://www.fattoasterfarm.com
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On Fri, Jun 7, 2013 at 3:19 PM, Frederick Horak <jacobflock at aol.com> wrote:

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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