Report Summary

28-Day Prechronic Toxicity, Biodistribution and Transgene Expression Study in Fischer 344 Rats for Single Submandibular Gland Injections of an Adenoviral Vector Carrying the Human Growth Hormone Gene (Ad-hGH)

Study Summary

Overall Study Design 
The management of diabetes and a number of other single protein deficiency diseases currently is achieved by the regular 
repeated injection of protein insulin for diabetes However gene therapy offers the opportunity for correcting the deficiency 
through the expression of a transgene coding for the required protein Co-administration of hydroxychloroquine HCQ 
redirected a significant portion of the hGH secretion from saliva into serum a more useful and desirable outcome for in vivo 
delivery of therapeutic proteins 
There has been little to no data published on toxicological effects in humans exposed to the adenoviral vector There is 
limited data published about toxicity in animals exposed to the adenoviral vector When male and female rats were 
administered a single cannulation dose of AdCMVhGH infusion and HCQ ip the growth hormone salivary gland extraction 
in rats resulted in higher antibodies in the saliva than in the blood serum 
The purpose of this study was to evaluate the response of F344 Rats associated with intraductal infusion of the 
submandibular gland with an adenoviral vector encoding the gene for human growth hormone hGH accompanied by the 
co-administration of hydroxychloroquine by intraperitoneal injection Distribution and availability of the transgene were 
evaluated by qPCR analysis in multiple tissues testisovary spleen liver lungs heart draining lymph nodes and the 
treated and contralateral untreated salivary gland The expression of the human transgene as detected by the presence of 
hGH in saliva and serum was also determined 

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