Abstract: This invention relates to compounds and their use as inhibitors or activators of Rab7 GTPase to treat or prevent the onset of Rab 7 GTPase-associated disorders such as neuropathies, cancer, metabolic diseases of bone and lipid storage. The invention is also applicable to infectious diseases where Rab7 is inactivated or its protein-protein interactions are modulated to facilitate intracellular survival of pathogens. The compound described acts as a competitive inhibitor of nucleotide binding and as such also has utility as a scaffold for targeting other small GTPases. In one aspect, methods of treatment of the invention are used to treat or prevent the onset of hereditary sensory neuropathies such as Charcot-Marie-Tooth type 2B disease. Related pharmaceutical compositions, assays, and drug screens are also provided.
Type:
Grant
Filed:
May 15, 2014
Date of Patent:
June 28, 2016
Assignee:
ST. UNM
Inventors:
Angela Wandinger-Ness, Larry Sklar, Jacob Agola, Zurab Surviladze, Jeffrey Aube, Jennifer Golden, Chad E. Schroeder, Denise S. Simpson
Abstract: A method of method of coating powdered medical agent onto a carrier particle for use in a dry powder inhaler may include applying ultrasonic energy to agglomerated powdered medical agent to deaggregate and aerosolize particles of the medical agent into particles having a desired average particle size, and coating at least one carrier particle with a desired amount of the deaggregated and aerosolized particles of the medical agent.