ISOA/ARF Drug Development Tutorial

ISOA/ARF Drug Development
Tutorial




By Jens Eckstein Institute for the Study of Aging Alzheimer Research Forum

TABLE OF CONTENTS

General Introduction ........................................................................................................... 2
Target Discovery—Overview............................................................................................. 2
Target Discovery—Disease Mechanism............................................................................. 3
Target Discovery—Disease Genes ..................................................................................... 3
Target Discovery—Target Type and “Drugability” ........................................................... 3
Target Discovery—Functional Genomics .......................................................................... 4
Target Validation—Overview ............................................................................................ 4
Target Validation—Knockout/in, Gain-of-Function, Transgenic Models ......................... 4
Target Validation—Pathways............................................................................................. 5
Target Validation—Clinical Data....................................................................................... 5
Target Validation—Antisense DNA/RNA and RNAi........................................................ 5
Target Validation—Chemical knockouts and Chemical ...