PgmNr D1385: Drug discovery in Drosophila.

Authors:
Tamy Portillo Rodriguez; Tom Hartl; Ethan Perlstein


Institutes
Perlstein Lab, PBC San Francisco, CA.


Keyword: drug discovery

Abstract:

Perlstein Lab is a public benefit corporation that discovers small molecule therapies for orphan diseases.  Since our conception in 2014, we have focused on the rare diseases Niemann Pick type C (NPC) and N-glycanase 1 (NGLY1) deficiency.  Drosophila with mutations in these disorders’ causal genes, npc1 and ngly1, are developmentally delayed during larval stages.  We will present our Drosophila high throughput drug screening platform and our progress on discovering small molecule modifiers of NPC and NGLY1 larval developmental delay.



Flybase Genetic Index:
1. FlyBase gene symbol: npc1a; FBgn: FBgn0024320
2. FlyBase gene symbol: pngl; FBgn: FBgn0033050