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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

The package insert for the drug, Elevidys , warns of adverse effects of acute liver injury and inflammation around the heart and muscles. million DNA bases. The DMD gene therapy delivers a shortened version of the dystrophin gene, just 4,558 DNA bases. He was treated on a Monday and died that Friday, as his organs shut down.

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Optimising AAV capsid purification through improved analytics

Drug Discovery World

This helps ensure a high concentration of loaded capsids and adequate removal of impurities, such as host cell proteins and DNA. The threat of empty capsids Capsids are the protein shell that would usually enclose the genetic material of a virus. DDW Volume 24 – Issue 1, Winter 2022/2023 References: Wright JF. doi:10.1038/GT.2008.65

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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.” At least, that’s what every textbook says.

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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.” At least, that’s what every textbook says.

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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. Read Switchable hydrophobic pockets in DNA protocells enhance chemical conversion. Nature Microbiology. Meeussen J.V.W.

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Codon Digest: Hackathon Prize Winners

Codon

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. A phage is a virus that infects bacteria.

DNA 52
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Codon Digest: Hackathon Prize Winners

Codon

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. A phage is a virus that infects bacteria.

DNA 52