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July 2019 Research Update

The Eyes Have It

Retinal disease can have devastating consequences for patients suffering from advanced glaucoma, atrophic macular degeneration, advanced diabetic retinopathy, and inherited retinal degenerations. These diseases affect millions of patients, but as their molecular etiologies are elucidated, novel gene therapy approaches for treatment have been proposed. For monogenic diseases, a defective or missing protein can be replaced by treatment with messenger RNAs (mRNAs) encoding the protein in question. Transient expression of transcription factors that up-regulate neuroprotective pathways could also be used to treat a variety of retinal diseases.

The design of effective and minimally immunogenic mRNAs is fairly well advanced. Although delivery of mRNAs has remained a challenge, lipid-nanoparticles (LNPs) are promising candidates for this process. Most studies have focused on mRNA delivery to the liver. Intravenous delivery of LNP-formulated mRNAs largely leads to liver targeting due to LNP binding of apolipoprotein E (Apo E) and subsequent uptake by abundant Apo E receptors on hepatocytes. While delivery to other tissues occurs...

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Zone In With The Zon Blog Post

How mRNA Therapeutics Are Entering and Revolutionizing the Monoclonal Antibody Field-Part 2-of-2

As indicated by the title, this blog is Part 2 of a two-part series on how mRNA therapeutics are entering the monoclonal antibody (mAb) field. Part 1 was posted on July 9, 2019, and it provides a historical perspective on the evolution...

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