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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. When I received my genetic test report in 2011, the world had never heard of base editing. The findings appear in Nature Medicine.

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Evolved prime editors are smaller and more efficient for therapeutic applications

Broad Institute

Their new editors are more efficient and specialized than previous versions, and are able to modify DNA in cultured cells and in animals that have been difficult to edit, including in immune system cells and inside the brain. In the new work, they focused on improving the heart of the prime editing system—the reverse transcriptase.

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DNA Analysis Finds New Target for Diabetes Drugs

NIH Director's Blog: Drug Development

Credit: Jane Ades, National Human Genome Research Institute, NIH Type 2 diabetes (T2D) tends to run in families, and over the last five years the application of genomic technologies has led to discovery of more than 60 specific DNA variants that contribute to risk. Plenge RM, Scolnick EM, Altshuler D. Nat Rev Drug Discov.

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Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

PLOS: DNA Science

The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. A Long and Winding Diagnostic Road The story begins in 2011, with Alessandra Biffi, MD, head of the clinical trial for MLD at the San Raffaele Telethon Institute for Gene Therapy in Milan. She passed away in 2013.

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CRISPR to Reveal How “Water Bears” (Tardigrades) Survive Extreme Environments

PLOS: DNA Science

The animals also tolerated conditions aboard the International Space Station in 2011, and seemed to have survived a crash into the moon from a probe in 2019. After all, they can reproduce even under radiation levels 1,000 times that which would kill a human, possessing admirable DNA repair skills.

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Addressing increasingly resistant drugs by infectious agents

Drug Target Review

5 Cytology-based profiling can facilitate antibiotic discovery efforts and, these assays assess cell cycle progression, nuclear and mitochondrial DNA content, mitochondrial DNA replication, nuclear DNA damage, mitochondrial membrane potential, and lysosome structure and function. link] Ojewale Kayode (2018). and Margaret, A.

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Merkin Prize in Biomedical Technology awarded to F. William Studier for development of widely used protein- and RNA-production platform

Broad Institute

I've always been interested in solving problems,” Studier told Brookhaven National Laboratory in a 2011 profile. Studier realized that the protein, called the T7 RNA polymerase, might be able to quickly and efficiently produce RNA from not only T7 DNA, but also from the genes of any organism. My interest is in basic research."

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