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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

CRISPRoff silences the targeted gene by adding methyl groups, chemical tags that prevent the gene from being transcribed or read into RNA and so from being expressed as protein. The first problem they had to tackle was the editor’s size, because the editor needs to be small enough to be packaged and delivered to specific cells in the body.

Disease 142
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Spectrum Solutions Secures Emergency Use Authorization (EUA) for its SDNA-1000 Saliva Collection Device, Expanding Access and COVID-19 Testing Options

The Pharma Data

Spectrum’s self-contained saliva collection kit provides critical sample consistency while suspending and neutralizing viral RNA transcripts completely inactivating the live virus. Qualified commercial RNA extraction chemistries include Perkin Elmer, Thermo Fisher, Roche, and Qiagen. Photo: Business Wire).

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Codon Digest: Seeing Colors After Gene Therapy

Codon

A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges. How do transcription factors couple up with the correct RNA molecule?

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Codon Digest: Seeing Colors After Gene Therapy

Codon

A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges. How do transcription factors couple up with the correct RNA molecule?

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Codon Digest: Injected Gene Editors

Codon

A giant dataset about the rat brain, and how neurons connect to each other, was collected and pooled together from 20 different research laboratories. Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). Nature Neuroscience. Gene Therapy.

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Janssen Announces U.S. FDA Approval of CABENUVA (rilpivirine and cabotegravir), the First Long-Acting Regimen for the Treatment of HIV

The Pharma Data

CABENUVA, a co-packaged kit with two injectable medicines, offers people living with HIV a new approach for maintaining viral suppression. In the ATLAS study, CABENUVA met the primary endpoint for noninferiority (the proportion of participants with plasma HIV-1 RNA ?50 50 c/mL, meeting noninferiority criteria.

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n-Lorem Foundation Preps First Doses to Treat Ultra-Rare Disease Patients for Free

The Pharma Data

It received FDA guidance January 5 th and is in the midst of preparing the first package of investigational new drug (IND) applications – usually with an n-of-1, for individual patients. That’s not the case with RNA-targeted drugs. Patient dosing is expected to begin later this year. “No

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