Broad Institute

JUNE 27, 2024

They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration. 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.

Disease 142

Disease Therapies Clinical Trials Engineering 142

Drug Target Review

APRIL 9, 2025

Now, his focus is on Gylden Pharma’s nanoparticle-based vaccine platform – a cutting-edge solution that bypasses the limitations of traditional RNA and live attenuated virus vaccines. In a recent interview, Professor Rademacher shared the key scientific breakthroughs and strategic insights behind this platform.

Virus 95

Virus Vaccine RNA Trials 95

Codon

NOVEMBER 1, 2024

Though my focus in this essay is narrow — I don’t discuss bottlenecks in clinical trials, human disease, or animal testing — I hope others will take on these challenges in similar essays. Live-seq similarly enables scientists to study RNA expression, without killing cells, over time. Subscribe to Asimov Press.

DNA 120

DNA RNA Molecular Biology Laboratories 120