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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

The treatment, which uses base editing to make a single-letter change in DNA, reduced levels of the disease-causing prion protein in the brain by as much as 60 percent. This has the potential to be a really promising strategy. But the researchers needed to deliver the base editors to the brain.

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

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. 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.

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Beyond Steel Tanks

Codon

In the case of semaglutide, those cells are Saccharomyces cerevisiae— also known as Baker’s yeast — engineered to secrete a peptide precursor that is later purified, chemically modified, packaged into an injectable or tablet form, and then shipped around the world. Continuing this method may not scale.

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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. Anyone who has tried to engineer a cell knows how tedious it can be.

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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. Anyone who has tried to engineer a cell knows how tedious it can be.

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

Agency IQ

Between 2012 and 2018, the five-year relative survival rate for patients with MM was 57%. The ideal scenario is to have a clinical trial that prospectively evaluates these endpoints, he said, providing a data package the FDA can use to evaluate such alternative endpoints.

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Analysis Chemical Thank You What we expect the EPA to do in April 2024

Agency IQ

The EPA is also proposing our response to a petition regarding the continued validity of EPA’s 2012 determination (and September 2017 affirmation) that participation in the Cross State Air Pollution Rule (CSAPR) meets the Regional Haze Rule’s criteria for an alternative to the application of source-specific BART.