“Gene Therapy to TPP1 Deficiency” Update

As many of you know, Blake’s Purpose has been working with Dr. Beverley Davidson out of the University of Iowa for about the past year or two.  We have funded a couple of grants for her and Dr. Katz (who is based in Missouri). Most recently we have been working with Bev on a her Gene Therapy study to TPP1 Deficient dogs.  In the past month or two we have had 2 very encouraging conference call updates with Bev on her progress and I wanted to share some of the exciting news. 

Within the past couple of years Bev has successfully treated mice and been able to reverse some of the deficits caused by TPP1 deficiencies; with that result in hand the Blake’s Purpose foundation has funded the next phase of her trial where she is attempting to reverse the effects of the disease in afflicted dogs by administering the dog colony with her meds using a couple of different approaches to see what works best.  The 1st dog was injected on April 30th and has been doing well, we are hoping to have some results in the fall regarding the dogs progress. 

We hold so much hope for what Bev is doing and pray that she is one step closer, if not the answer to finding a cure for these children.  Our most recent conversation with Bev was so encouraging; leading us to think that if all goes well with her current projects then the next step is to produce a clinical grade vector and try and get these meds to trial in LINCL children. This next phase of work will undoubtedly cost a lot of money/upwards of a million dollars which is another reason why we are trying to step up our fundraising.  If not for hope, what do we all have?

This entry was posted in Uncategorized. Bookmark the permalink.

Comments are closed.