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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My

Disease 80
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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.

Disease 144
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A Look Back

Dark Matter Blog

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. Synta was focused on small molecule drug discovery and development in oncology and inflammatory diseases. I wanted to use molecular biology to create drugs.

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Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

Cell-based assays , while more complex to develop and use, offer another option for characterizing product activity at a slightly higher level, allowing sponsors to measure the further downstream effects of products (e.g., morphological cell changes or alterations in protein production or expression).

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Cave Coronavirus in Wuhan Lab Seeded COVID – The Truth Has Always Been Out There, in the Genetics

PLOS: DNA Science

Experiments at the Wuhan facility were done at the lowest two of the four standard biosafety levels, which were established at the dawn of recombinant DNA research in the 1970s. And a change to one specific part of the virus enabled it to locate and glom onto our cells, then slip inside and take over the protein production line.

Virus 52
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Messenger RNAs with multiple “tails” could lead to more effective therapeutics

Broad Institute

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.

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Codon Digest: Bacteria Resist Every Virus

Codon

Digital nanoreactors to control absolute stoichiometry and spatiotemporal behavior of DNA receptors within lipid bilayers. Peroxisomal metabolic coupling improves fatty alcohol production from sole methanol in yeast. Optogenetic closed-loop feedback control of the unfolded protein response optimizes protein production.

Virus 52