Another gene-directed strategy that has been tested with positive results is the use of short hairpin RNAs (shRNAs) targeting mutant ATXN3 [283]
A peptide is a chain of amino acids held together by peptide bonds, and those bonds are precisely what digestive enzymes (pepsin, trypsin) are built to cut, with the acidic stomach environment finishing the job so an oral peptide is largely destroyed before it can be absorbed, which is why injection is the default
This human safety record however limited in scope is a meaningful differentiator when researchers evaluate the preclinical-to-translational potential of synthetic peptides
Generated ROS can seriously harm biological components like lipids and nucleic acids, which have a clear cytotoxic effect on cancer cells (Nelson et al., 1987)
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