Endocrine Abstracts

The growth hormone receptor antagonist was discovered via expression of mutated GH genes in transgenic mice. Although we were attempting to generate potent GH analogs, our mouse studies indicated that the compound was ‘inhibiting’ GH action. We subsequently showed that it was a GH receptor antagonist, the first of its type. Many hurdles needed to be ‘jumped’ in the development of this compound into a drug including protection of intellectual property; fundi...

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...