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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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AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012).

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Day Zero Antivirals for Future Pandemics

Codon

When COVID-19 emerged in 2019, by contrast, mRNA vaccines developed by Pfizer and Moderna took just 326 days from the initial sequencing of the virus to gaining approval for emergency use. These drugs would be ideal tools to bridge the wait for a vaccine against a quickly-spreading virus.

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Driving Toward Nanopores

Codon

And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.

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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. million DNA bases. The DMD gene therapy delivers a shortened version of the dystrophin gene, just 4,558 DNA bases. I was overoptimistic.

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Addressing the Challenge of Antibiotic Resistance

DrugBank

Their joint efforts have led to the development of gepotidacin , a novel first-in-class antibiotic that inhibits the bacterial enzymes topoisomerase II and DNA gyrase, which are essential for DNA replication and repair. For instance, the U.S.

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Codon: Notes on Progress #2

Codon

“The recombinant DNA breakthrough has provided us with a new and powerful approach to the questions that have intrigued and plagued man for centuries. The central dogma is often depicted as DNA→RNA→protein, but it’s much more: A biophysical marvel inside the smallest of vessels. Biology is a Burrito.

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