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Cell and gene therapy development moves into cardiac indications

Drug Target Review

Viral vectors, such as adeno-associated viruses (AAVs), are commonly used to deliver therapeutic genes to cardiac cells. RNA-based therapies, including mRNA and RNA interference (RNAi), are being explored to modulate gene expression in heart cells.

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Codon Digest: CAR-T Therapy for Neuroblastoma

Codon

Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. The RNA strands shut down a specific gene in the insects, and killed half after 7 days. RNA may offer a safer form of pest control in the future. Nature Chemical Biology. Current Biology.

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Codon Digest: Injected Gene Editors

Codon

Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). By combining this new variant with standard sequencing methods, it’s possible to identify RNA modifications in a really simple way. Communications Biology. Ricciardi M.J.

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Codon Digest: Vaccine Printer Go Brrrrr!

Codon

This works for many different cell types, and the method can also be used to deliver several different gene editors at once. It’s an easier way to get CRISPR into cells! Other methods to deliver Cas9 (like with viruses) are way less efficient, and seem to vary wildly from one cell to the next. Nature Cell Biology.

Vaccine 52
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Codon Digest: Vaccine Printer Go Brrrrr!

Codon

This works for many different cell types, and the method can also be used to deliver several different gene editors at once. It’s an easier way to get CRISPR into cells! Other methods to deliver Cas9 (like with viruses) are way less efficient, and seem to vary wildly from one cell to the next. Nature Cell Biology.

Vaccine 52
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Levers for Biological Progress

Codon

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. Even more fundamentally, a virtual cell is a way to encapsulate the current extent of our biological knowledge. Even the humble E.

DNA 105
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Codon Digest: GPT-4 Controls a Robot

Codon

Compact gene editors , called enOsCas12f1 and enRhCas12f1, are smaller than many existing Cas proteins and can thus be more easily delivered in a single virus for gene therapies. By engineering the sgRNA and optimizing these proteins, new variants had high editing efficiencies and low off-target effects in human cells. Cell Reports.