CRISPR-Cas9 tagged posts

DNA Damage Repaired by Antioxidant Enzymes

In crisis, the nucleus calls antioxidant enzymes to the rescue. The nucleus being metabolically active is a profound paradigm shift with implications for cancer research.

Summary points

The human nucleus is metabolically active, according to the findings of a new study in Molecular Systems Biology by researchers at the CRG in Barcelona and CeMM/Medical University of Vienna,

In a state of crisis, such as widespread DNA damage, the nucleus protects itself by appropriates mitochondrial machinery to carry out urgent repairs that threaten the genome’s integrity

The findings represent a paradigm shift because the nucleus has been historically considered to be metabolically inert, importing all its needs through supply chains in the cytoplasm

Cancer hijacks cellular metabolism...

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Scientists develop a Cancer Vaccine to Simultaneously Kill and Prevent Brain Cancer

Scientists develop a cancer vaccine to simultaneously kill and prevent brain cancer
Scientists developed a bifunctional therapeutic strategy by transforming living tumor cells into a therapeutic. Shah’s team engineered living tumor cells using the gene editing tool CRISPR-Cas9 and repurposed them to release tumor cell killing agent. In addition, the engineered tumor cells were designed to express factors that would make them easy for the immune system to spot, tag and remember, priming the immune system for a long-term anti-tumor response. The team tested their repurposed CRISPR-enhanced and reverse-engineered therapeutic tumor cells (ThTC) in different mice strains including the one that bore bone marrow, liver and thymus cells derived from humans, mimicking the human immune microenvironment...
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New CRISPR-based Tool Inserts Large DNA Sequences at Desired Sites in Cells

The arm of lab scientist plucks a segment of a DNA strand.
Caption:Building on the CRISPR gene-editing system, MIT researchers designed a new tool that can snip out faulty genes and replace them with new ones.
Credits:Image: MIT News, with images from iStockphoto

Building on the CRISPR gene-editing system, MIT researchers have designed a new tool that can snip out faulty genes and replace them with new ones, in a safer and more efficient way.

Using this system, the researchers showed that they could deliver genes as long as 36,000 DNA base pairs to several types of human cells, as well as to liver cells in mice. The new technique, known as PASTE, could hold promise for treating diseases that are caused by defective genes with a large number of mutations, such as cystic fibrosis.

“It’s a new genetic way of potentially targeting these reall...

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Restoration of Retinal and Visual Function following Gene Therapy

Base editing restores vision
A mutation in an inherited blindness mouse model abolishes the expression of RPE65, a key enzyme in a visual cycle, in the RPE cells. Base editing treatment can correct the mutation and restore functional RPE65 (green), thereby restoring vision in mice

New generation CRISPR technology lays foundation for therapeutics to treat a wide range of inherited ocular diseases. A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, “Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing,” illustrates the use of a new generation CRISPR...

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