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

Broad Institute

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142
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What to expect from PEGS Europe 2023: Day 1

Drug Discovery World

The luncheon presentation will be done by James Keck, PhD, President’s Innovation Fellow and Senior Director, Innovation and Product Development, Product Development, The Jackson Laboratory. CCO & Co-Founder, ATUM, on: “Using machine learning to predict protein expression”. Claes Gustafsson, Ph.D,

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Codon Digest: CAR-T Therapy for Neuroblastoma

Codon

Marine sponges were cultured in the laboratory continuously for the first time. AI + Bio *Transfer learning for cross-context prediction of protein expression from 5’UTR sequence. Read Rational design of a highly immunogenic prefusion-stabilized F glycoprotein antigen for a respiratory syncytial virus vaccine.

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Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

The Pharma Data

In addition, the observed median PFS shows that the regimen can induce a sustained and durable response, which is also shown consistently by the induction of a specific immune response. This response developed as early as D43 and sustained at 6 months after initiation of therapy, consistent with the durable disease-control.

Vaccine 52