Researchers at the Massachusetts Institute of Technology (MIT) have developed an approach that uses experimental chemistry and artificial intelligence to facilitate delivery of a drug to treat Duchenne muscular dystrophy (DMD).
The approach aims to determine which cell-penetrating peptides can be attached to antisense phosphorodiamidate morpholino oligomers (PMO) to help permeate the cell nucleus to modify a mutated dystrophin gene and enable production of a key protein typically missing in patients with DMD.
The researchers built a library of 600 miniproteins, each attached to PMO, by mixing and matching 57 peptides, and quantified each miniprotein's ability to deliver drugs across the cell.
MIT's Rafael Gomez-Bombarelli said, "The key innovation is using machine learning to connect the sequence of a peptide, particularly a peptide that includes non-natural amino acids, to experimentally-measured biological activity."
The sequences proposed by the model were found to be more effective than previously known variants.
From MIT News
View Full Article
Abstracts Copyright © 2021 SmithBucklin, Washington, DC, USA