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Enhancing gene therapy with Circio

Drug Target Review

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. Cas9 binds and uses RNA as an address-tag. The AI-designed blocking protein was the cargo of the dCas9-RNA construct.

RNA 52
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Reinventing the small molecule toolbox: from proteins to RNA

Dark Matter Blog

Prior to 2015, I had a casual relationship, at best, with targeting RNA. Targeting RNA is a Whole New World Then in 2015, I became smitten and eloped with RNA, setting out to build a company devoted to bringing to bear industrial drug discovery concepts and methods on a new problem of drugging RNA with small molecules.

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Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

In the new work published today in Nature Biotechnology , the team adapted engineered virus-like particles (eVLPs) that they had previously designed to carry base editors — another type of precision gene editor that makes single-letter changes in DNA.

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Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

DNA 106
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Codon Digest: Seeing Colors After Gene Therapy

Codon

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” LEGO robot to make sucrose gradients.

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Codon Digest: Seeing Colors After Gene Therapy

Codon

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” LEGO robot to make sucrose gradients.