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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 October 4, 2022. appeared first on DNA Science.

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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. In this experiment, however, the cutting function of the Cas9 protein is disabled so the genomic DNA sequence is unaltered.

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Ensuring reliable assays: Why the ideal microplate is an invisible one

Drug Discovery World

But it comes with surface charge that will absorb proteins, DNA, and other things, unless you do something to treat that surface. Everything is packaged in antistatic bags to dissipate any static that might become relevant. We pay great attention to our mould design, which has a lot to do with consistency in performance.

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Article FDA Thank You FDA’s oncology advisory committee to consider MRD as a multiple myeloma endpoint

Agency IQ

Janssen’s Carvykti (ciltacabtagene autoleucel, or cilta-cell) then received approval in February 2022 for treatment of adult patients with RRMM and the same treatment history as that required for treatment with ide-cel. However, the process of preparing and delivering these therapies takes time.

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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: 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.