Approved Research

Prediction of novel enhancement-targeting protein chimera target specificity from underlying genetic variability.

Lay summary

New advances in science permit new types of medicines to be made. We found one such advance, which uses a human cell's normal function to make a special type of protein that works better than regular proteins. However, we want to ensure that our enhanced proteins are safe, specific, and have low potential for harmful side-effects.

To that end, we will use the requested data to address the following aims:

Aim 1: Identify DNA-level changes that are specifically related with a particular disease or human health outcome, because these are likely to be the safest for treating with our enhanced proteins.

Aim 2: Determine which DNA changes are likely to make people more susceptible to disease, or to impact how well they respond to medications.

Aim 3: Elucidate how treating DNA changes with our enhanced proteins might result in undesirable side-effects.

Scientific rationale: Previously, most targeted therapies have focused on inhibiting over-active proteins. However, little attention has been given to activating under-active proteins. This leaves a whole large area of treatment types unexplored. We identified a new protein enhancing approach that will allow us to make three new kinds of medicines: 1) increasing the amount of proteins when there aren't enough, 2) moving proteins to the correct place if they're lost, or 3) making "lazy" proteins work harder if they're not doing enough at baseline.

We expect this work will have meaningful benefit for a broad range of diseases, including but not limited to: respiratory diseases (i.e. asthma, COPD), neuro and musculoskeletal degeneration (dementia, Parkinson's, stroke, motor-neuron disease), cardiac disease, organ failure, immune dysfunction, endocrine & metabolic disorder, vision and hearing impairment, gestational and puerperium related illness, and congenital disruptions and chromosomal abnormalities.