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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

Therapies 162
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What Limits a Genome’s Size?

Codon

And what they found, remarkably, is the world’s largest genome ; each fern cell contains 160.45 billion bases of DNA which, if unfurled, would extend for more than 100 meters—taller than the Statue of Liberty. billion bases of DNA, the human genome measures just 2 meters in length when stretched end-to-end.

DNA 62
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Delivering on the promise of gene editing

Drug Discovery World

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Reduce/eliminate chromosomal disruptions caused by double-strand DNA breaks.

DNA 130
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Codon Digest: Injected Gene Editors

Codon

But now, by studying DNA extracted from microbes in the blood of almost 10,000 healthy people, this paper shows that there is no such thing. Read Transcription factors bind to DNA and control gene expression. They tend to “group up” in cells. Molecular Systems Biology. Nature Microbiology. Meeussen J.V.W.

DNA 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. From Zhang et al.

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. From Zhang et al.

Vaccine 52
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Codon Digest: CAR-T Therapy for Neuroblastoma

Codon

.” This technology could be used to design protein therapeutics that can bind to, and “shut down,” harmful or misfolded proteins in living cells. Thousands of transcription factors — proteins that bind DNA and control gene expression — were studied in human cells. Nature Cell Biology.