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Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. Like Cas9, ZFPs can serve as guide proteins to direct the tool to a target site in DNA.
To achieve an aggressive timeline from DNA to investigational new drug (IND) application, drug developers have strengthened collaborations with contract development & manufacturing organisations (CDMOs) to expedite drug development. FDA no longer has to require animaltesting for new drugs. References: 1 Wadman M.
Mice raised at five different animal facilities in Europe, under otherwise identical conditions , have “persistent differences in body weight” and behavior. There are even differences in how their genomic DNA is packaged inside of neurons. DNA differences accumulate over generations of breeding.
Though my focus in this essay is narrow — I don’t discuss bottlenecks in clinical trials, human disease, or animaltesting — I hope others will take on these challenges in similar essays. Perhaps you’ve seen this chart before, which shows the falling costs of DNA sequencing over the last two decades.
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