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NIH Phase-I Trial: Aquaporin Gene Therapy

Started by markt, May 03, 2019, 09:51:52 AM

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markt

https://www.nidcr.nih.gov/news-events/nidcr-news/pioneering-gene-therapy-dry-mouth

Recruitment for the first in human gene therapy for Sjogrens dry mouth/eyes starts this fall.  This builds on successful in human trials done for radiation patients who have had glandular epithelia/acinar cells destroyed by irradiation.  Proof of concept studies have been completed/published for Sjogrens...and now this therapy is being matured for adaptation to clinical practice. 

POC info for NIH Sjogrens/SG disease unit can be easily googled.  I did a study protocol with them in Bethesda last month...I was interested to learn how much they collaborate with John's Hopkins (why I went up there originally).

FDA should post the above study protocol shortly.

Mark

markt

http://perirx.com/wp-content/uploads/Sjogrens-Quarterly-Prospects-for-Genetic-Intervention-in-the-Salivary-Glands-of-Sjogrens-Patients-.pdf

More background info on the subject trial for those interested.  The article is from 2015, so obviously a a lot of water under the bridge since this was published.