Molecular Diagnostics Lead To Remedies That Interfere with Pathways Upstream from Protein Synthesis

The Economist March 14th, 2020 pp4-8 Technology Quarterly|Personalised medicine

Congenital disease. “Broken” “Damaged genes can reveal a lot about human biology”

Molecular genetics, makes reaching a diagnosis faster and more accurate, yields new information about disease mechanisms but until now didn’t lead to improved treatments. Reducing the time to diagnosis from years to months ends the “diagnostic odyssey” and having an accurate diagnosis helps deploy the appropriate treatment. “Sometimes, the correct care has dramatic results” allowing caregivers to appropriately adjust diet or treatments. Having a better understanding of molecular mechanisms allows researchers to specifically target research for cures.

In the past, nearly all pharmaceutical therapies focused on correcting defects caused by an altered protein or by abnormal protein levels. Today, molecular remedies are targeting upstream of protein synthesis by interfering in the conversion of DNA to mRNA and mRNA to protein. Companies like “Maze Therapeutics….is looking for ‘genetic-modifiers’ that alter the course of conditions like ALS”. An example of a genetic-modifier is antisense oligonucleotides (ASOs). “The fact that ASOs are based on sequences means that they can be ‘programmed’ to inhibit the synthesis of a wide range of proteins. An ASO can also be designed and used to treat a disease unique to a single patient-what doctors call an ‘n of 1 trial’”. While able to create treatments even for rare diseases the developmental costs are huge. Start-ups and a number of organizations are working to fund these efforts including Mila’s Miracle Foundation, Rare As One Network and The n-Lorem Foundation.

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