Re: PAH Plans get a write up in Pulmonary Hypertension News
in response to
by
posted on
May 21, 2019 11:53AM
"I’m beginning to get the idea that Apabetalone, and/or any subsequent/future enhancements or modifications thereto, could have a positive impact on almost any medical condition that has a genetic implication or component to it."
I can tell you are really excited about the potential of apabetalone and other epigenetic drugs. However, keep in mind that apabetalone, via interfering with BET bromodomain binding to acetyl-lysines on histones, affects the mRNA transcription of genes encoded by the DNA. Apabetalone does not change the DNA sequence. It doesn't fix mutations. It doesn't edit genes like CRISPR. Most genetic diseases result from point mutations in the DNA or DNA insertions/deletions that affect the activity, function, stability, or cellular localization of the encoded protein due to changes in the encoded protein amino acid sequence. Apabetalone and other epigenetic drugs are not going to fix these abnormalities in the DNA sequence that result in the abnormal protein level or function. Some DNA mutations in non-coding (non-exon) regions of the DNA (i.e. promoter, 3' UTR, introns, "junk" DNA between genes) may affect the level of a gene's mRNA expression level without affecting the encoded protein amino acid sequence. For these non-coding mutations that may cause human disease due to abnormal overexpression of a mRNA, I could definitely see potential for BET inhibitors and other epigenetic drugs to silence/cool-off/downregulate the expression of these overexpressed mRNA back to a more normal, healthy level.
BearDownAZ