3 ms·
I would like to know of any diseases which are successfully treated by the techniques described above. Have there ever been any certifiably broken biological pa
by jganetsk 15y ago
I would like to know of any diseases which are successfully treated by the techniques described above. Have there ever been any certifiably broken biological pathways, indicated through genetics, that have been corrected through drug therapy? It seems like most drugs have very broad effects and don't adjust an actually diseased pathway. Even monoclonal antibodies for inflammatory diseases seem to be a poor match for the specific treated diseases.
- sungam 15y agoA large fraction of modern drug development is focused on specific biochemical pathways, often identified via the study of rare genetic disorders or genome-wide association studies. For example, patients with Familial hypercholesterolemia, a relatively rare genetic disorder, provided strong evidence for a causal role of cholesterol in ischaemic heart disease (a discovery for which Brown and Goldstein were awarded the Nobel prize). The enzyme inhibited by statins (HMG-CoA-reductase) is a key player in this pathway.