ECE2022 Poster Presentations Pituitary and Neuroendocrinology (127 abstracts)
Gene therapy of growth hormone resistant dwarfism in the laron mouse model – comparison of two doses
Kian Chuan Sia 1 , Shu Uin Gan 1 , Siti Humairah Mohd Rodhi 1 , John J. Kopchick 2 , Michael J. Waters 3 & Kok Onn Lee 1
1National University of Singapore, Singapore, Singapore; 2Ohio University, United States; 3The University of Queensland, Australia
Growth hormone receptor (GHR) defective Laron Syndrome (LS) has been treated with daily subcutaneous recombinant insulin-like-growth factor 1 (IGF1) injections lasting many years. We have reported the results of treatment of 4-5 week old Laron dwarf mice (GHR-/-) with a single injection of an adeno-associated virus vector with a murine GHR (AAV-GHR) and a liver specific promoter at a dose of 4 × 1010 vector genome per mouse (Sia et al, Gene Therapy 2022).
Methods: In the present study, we report the results of increasing the dose to four times our earlier reported dose: 16 X 1010 vector genome per mouse. In both groups of mice, low dose (previous 4 X 1010) and high dose were injected at the same age of 4-5 weeks. Mice injected with the same doses of AAV-Luciferase were used as controls. The mice were monitored for a total of 20 weeks, after which they were euthanized. The organs were harvested and weighed. Serum was obtained for analyses.
Results: The serum growth hormone (GH) levels decreased, and IGF1, IGF binding protein 3 (IGFBP3) and acid labile subunit (ALS) increased compared to the respective controls. There was a significant increase in body weight and length with both doses, but the difference between the two doses was not statistically significant. All the major organs increased in weight and size, but there was no significant difference between the 2 doses.
Conclusion: Surprisingly, increasing the single dose by four times did not result in a significant increase in the response.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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