The Open Targets Blog

JANUARY 30, 2025

gget ( [link] ) is a free, open-source command-line tool and Python package designed to enable efficient querying of large genomic databases, such as Ensembl, UniProt, and NCBI. Joe is a fourth-year USC-Caltech MD-PhD student in the Pachter Lab, and he is working on a novel algorithm to detect carcinogenic variants in RNA sequencing data.

Bioinformatics 74

Bioinformatics Immune Response RNA Packaging 74

Codon

JUNE 5, 2025

The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. pyogenes protein — whose compactness makes them far easier to package into viral vectors and deliver into the human body. Collectively, these repeat-protospacer regions are known as CRISPR arrays.

DNA 81

DNA RNA Clinical Trials Engineering 81

Broad Institute

JUNE 27, 2024

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

Disease Therapies Clinical Trials Engineering 142

Codon

APRIL 2, 2024

Like, if we start thinking about therapeutic circuits, how can we design the components in such a way that they are not going to cause an immune response? The RNA Exporter , which was developed by Felix Horns and others in my lab, is one example. The exported RNA could transfect just the target cell.

RNA 58

RNA Engineering Molecular Biology Synthetic Chemistry 58

The Pharma Data

DECEMBER 16, 2020

Gustave Perna, who oversees the logistics for Operation Warp Speed, told reporters earlier this week that McKesson will package and distribute the vaccine to more than 3,000 sites across the country. The Moderna vaccine is similar to Pfizer and BioNTech’s vaccine in the sense that they both use an approach utilizing messenger RNA (mRNA).

Vaccine 52

Vaccine Immune Response FDA Virus 52

Codon

JULY 27, 2023

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?

Therapies 52

Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

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?

Therapies 52

Therapies RNA DNA Engineering 52