Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor

摘要

Described herein is a system which uses a gene therapy particle that includes at least one gene, cDNA, fragment or analogue of at least one neurotropin that binds to the trkB receptor or the trkB receptor itself. The gene therapy particle is capable of being delivered to a subject in need thereof for therapy of a metabolic disorder. In a particular aspect, this invention is directed to an inventive method that demonstrates a remarkable efficacy on lowering body weight.

主权项

1. A gene therapy particle comprising:
a first expression cassette comprised of a heterologous promoter operatively linked to a nucleotide coding sequence (cDNA) encoding a brain derived neurotrophic factor (BDNF) which is linked to WPRE; and, at least a second expression cassette comprised of cDNA AGRP484 nt 568-1051 in SEQ ID NO:7) linked to miR-Bdnf which is, in turn, linked to WPRE74 having SEQ ID NO:3.