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Breakthrough in gene editing

Breakthrough in gene editing

Science

h

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https://phys.org/news/2019-06-gene-editor-harnesses-genes-precise.html

"...With INTEGRATE, a set of enzymes can carry out the entire DNA integration process, reliably inserting an arbitrary DNA payload into a precise location within a cell's genome, without relying on the host cell's DNA repair machinery...."

The ability to reliably control exactly were a large new gene is inserted into the genome is a first in genetic engineering and should revolutionize genetic engineering. I say this is excellent work and is exactly what was needed!

"...The technique should enable a vast range of new gene editing opportunities. Many biotechnology products, including gene and cell therapies, engineered crops, and biologics, require precise integration of large genetic payloads.

The INTEGRATE technology offers a fresh new approach with the same programmability and ease of use as CRISPR-Cas9, but without the adverse effects associated with DNA breaks.

"We can program this CRISPR-transposon system to integrate its genetic payload at virtually any genomic site, and by understanding how it works, we will be able to engineer it to be even more effective,"...
Sternberg's team developed the INTEGRATE technology using bacterial genetics experiments, and they are now testing it in additional cell types, including mammalian cells.

Based on the developmental trajectory of CRISPR-Cas9 technology, Sternberg says there's good reason to believe that precise DNA integration will work as effectively in mammalian cells as it does in E. coli, opening up the door to basic research uses and eventual clinical applications..."

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Just think; If you can ability to reliably control exactly were a large new gene is inserted into the genome of a HUMAN, you can replace a defective gene with the undefective gene and instantly cure him of a horrible genetic disease!

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@humy said
Just think; If you can ability to reliably control exactly were a large new gene is inserted into the genome of a HUMAN, you can replace a defective gene with the undefective gene and instantly cure him of a horrible genetic disease!
Efficiency, heterogeneity and off-target effects are still a big problem.

But.... in some cases it still works.

https://www.nejm.org/doi/full/10.1056/NEJMoa1609677

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@humy said
Just think; If you can ability to reliably control exactly were a large new gene is inserted into the genome of a HUMAN, you can replace a defective gene with the undefective gene and instantly cure him of a horrible genetic disease!
How do you get that change into every cell in the body? That is what I wonder about.
It seems obvious you can't do it just one cell at a time, there has to be some batch process to do all the cells.

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