Biodistribution and Expression Study

Approximately 200 mice will be placed into 5 groups and given (intravenously, IV) different levels of the GNE lipoplexes. The mice will be observed by animal technicians each week for 90 days for physical changes such as weight, temperature, and stress. At the end of the study, blood as well as 10 major organs will be collected to see how, when, and where the GNE lipoplexes affect the body. A trained pathologist will then review the organs and report on the microscopy anatomy of the cells (histology). The cost for the animals, housing, materials and equipment, technicians, and pathologist review is approximately $150,000.

Molecular Analysis

Once all of the blood and organs are harvested, the tissues will be analyzed by Gradalis. DNA, RNA, protein and sialic acid levels will be tested in all of the tissues collected. This analysis will show how, when, and where the GNE lipoplexes affect the mice. The cost for the molecular analysis (including time, equipment, materials and reagents) is approximately $120,000 per month. It is predicted that it will take approximately 4 months to conduct these analyses.

Safety and Toxicology Study

In this study approximately 350 rodents will be given (intravenously, IV) 3 different doses of the GNE lipoplexes. The mice will be observed by animal technicians each week for 90 days for physical changes such as weight, temperature, and stress. At the end of the study, blood as well as 33 major organs will be collected. The blood collected will be analyzed for a complete blood count (CBC), coagulation, and chemistry (reveals information on a variety of organs and body systems). A trained pathologist will review the organs and report on the microscopy anatomy of the cells (histology). The cost for the animals, housing, materials and equipment, technicians, and pathologist review is approximately $570,000.

The animal studies will be conducted at an animal facility with oversight by an Institutional Animal Care and Use Committee
(IACUC).

More than anything, I hate to ask for donations because of all the support I have already received from all of you. However, many people have asked if we still need money for the HIBM research. The answer is �yes.�We still need donations to support the dose escalation trial I am participating in now, as well as the phase 1 trial that will hopefully begin following the dose escalation trial. Phase 1 trial will include nine more patients. Thank you so much for the donations you have already given, and please only donate if you feel lead to do so.