article thumbnail

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

Broad Institute

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.

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.

Insiders

Sign Up for our Newsletter

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

article thumbnail

Reinventing the small molecule toolbox: from proteins to RNA

Dark Matter Blog

Prior to 2015, I had a casual relationship, at best, with targeting RNA. Targeting RNA is a Whole New World Then in 2015, I became smitten and eloped with RNA, setting out to build a company devoted to bringing to bear industrial drug discovery concepts and methods on a new problem of drugging RNA with small molecules.

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.

Therapies 137
article thumbnail

Cultivated Meat? Let Them Eat Snake

PLOS: DNA Science

Biotechnology has solved many problems, from recombinant DNA and monoclonal antibody-derived drugs, to gene therapy and stem cell transplants, to RNA-based vaccines and genetically modified plants that resist diseases and pesticides. We package them into a range of branded consumer products.” ” Why Did It Have to be Snakes?

DNA 98
article thumbnail

Scientists map the locations of hundreds to thousands of cell types across a mammalian brain

Broad Institute

The studies are part of a package of 10 papers in Nature that take distinct, yet complementary, approaches to mapping the mouse nervous system at the single-cell level. The package also includes the spatial, single-cell atlas of the mouse brain and spinal cord that was led by Wang and Liu and was first published online in September in Nature.

DNA 140
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. RNA-guided transcriptional silencing in vivo with S. DNA encodes the basic building blocks of life and the epigenome controls which blocks are accessible for active use. Molecular Therapy.

DNA 106