Endocrine Abstracts

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 202...