Remove Engineering Remove Packaging Remove RNA
article thumbnail

Case study: gget’s new Open Target module

The Open Targets Blog

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.

article thumbnail

A Visual Guide to Genome Editors

Codon

The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. This article provides a summary of the major CRISPR systems, 2 including the naturally occurring CRISPR-Cas9, -Cas12, and -Cas13 systems, as well as base editors, prime editors, and the recently uncovered bridge RNA system.

DNA 81
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Levers for Biological Progress

Codon

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. SeqFISH, developed by Long Cai’s group at Caltech, maps the spatial positions of thousands of RNA, DNA, or protein molecules within single cells.

DNA 132
article thumbnail

Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.

article thumbnail

Cas9 ancestor engineered into a compact genome editing tool

Broad Institute

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells.

article thumbnail

New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor. This engineered capsid opens up a world of possibilities."

Therapies 137
article thumbnail

Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The molecular machinery of the epigenetic system can selectively package specific regions of DNA away, making them inaccessible and less active. This platform opens the door to treating complex multigenic diseases, effective tissue engineering, and fine-tuning immune cell differentiation. Molecular Therapy. 2021;29(9):2640–59.

DNA 106