Sometimes the science impresses you more than at other times but give it up for the sciencers who really did restore some sight to blind rats. Probably coming as no surprise, they did it with genome modification. (Science Daily: New gene-editing technology partially restores vision in blind animals)
Until now, techniques that modify DNA -- such as the CRISPR-Cas9 system -- have been most effective in dividing cells, such as those in skin or the gut, using the cells' normal copying mechanisms. The new Salk technology is ten times more efficient than other methods at incorporating new DNA into cultures of dividing cells, making it a promising tool for both research and medicine. But, more importantly, the Salk technique represents the first time scientists have managed to insert a new gene into a precise DNA location in adult cells that no longer divide, such as those of the eye, brain, pancreas or heart, offering new possibilities for therapeutic applications in these cells.
- Science Daily
A neuron or nerve cell is one which does not divide anymore and you know already those are the kind which don't come back if they're damaged or destroyed. Using CRISPR, it's potentially possible to fix them.
"We were able to improve the vision of these blind rats," says co-lead author Reyna Hernandez-Benitez, a Salk research associate. "This early success suggests that this technology is very promising."
The team's next steps will be to improve the delivery efficiency of the HITI construct. As with all genome editing technologies, getting enough cells to incorporate the new DNA is a challenge. The beauty of HITI technology is that it is adaptable to any targeted genome engineering system, not just CRISPR-Cas9. Thus, as the safety and efficiency of these systems improve, so too will the usefulness of HITI.
"We now have a technology that allows us to modify the DNA of non-dividing cells, to fix broken genes in the brain, heart and liver," says Izpisua Belmonte. "It allows us for the first time to be able to dream of curing diseases that we couldn't before, which is exciting."
"Using this NHEJ pathway to insert entirely new DNA is revolutionary for editing the genome in live adult organisms," says Keiichiro Suzuki, a senior research associate in the Izpisua Belmonte lab and one of the paper's lead authors. "No one has done this before."
Until now, techniques that modify DNA -- such as the CRISPR-Cas9 system -- have been most effective in dividing cells, such as those in skin or the gut, using the cells' normal copying mechanisms. The new Salk technology is ten times more efficient than other methods at incorporating new DNA into cultures of dividing cells, making it a promising tool for both research and medicine. But, more importantly, the Salk technique represents the first time scientists have managed to insert a new gene into a precise DNA location in adult cells that no longer divide, such as those of the eye, brain, pancreas or heart, offering new possibilities for therapeutic applications in these cells.
- Science Daily
A neuron or nerve cell is one which does not divide anymore and you know already those are the kind which don't come back if they're damaged or destroyed. Using CRISPR, it's potentially possible to fix them.
"We were able to improve the vision of these blind rats," says co-lead author Reyna Hernandez-Benitez, a Salk research associate. "This early success suggests that this technology is very promising."
The team's next steps will be to improve the delivery efficiency of the HITI construct. As with all genome editing technologies, getting enough cells to incorporate the new DNA is a challenge. The beauty of HITI technology is that it is adaptable to any targeted genome engineering system, not just CRISPR-Cas9. Thus, as the safety and efficiency of these systems improve, so too will the usefulness of HITI.
"We now have a technology that allows us to modify the DNA of non-dividing cells, to fix broken genes in the brain, heart and liver," says Izpisua Belmonte. "It allows us for the first time to be able to dream of curing diseases that we couldn't before, which is exciting."
- Science Daily
The first concern in any genetic science is whether the modification will ultimately become part of the genome but the candidates for this type of medicine are likely older and probably past the age of reproduction. Usually any GMO applies to germ cells which grow into adult organisms but this technique works on the adults.
- Science Daily
There's more detail in the article about the process but the above shows the concept and the result.
Get accustomed to hearing of CRISPR since that name pops up frequently. If you will be a modern day gene-splicing cowboy / cowgirl, it seems there's a good chance CRISPR is in your future.
There is deep Rockhouse reservation about modifications to the human genome going out into the open gene pool to become Homo sapiens trumpus, a simply better human or at least a louder one. That goes out into a whole raft of already-written or easily-predictable sci-fi horror stories.
Ed: how about 'open source humans?'
Not a problem since the techniques for CRISPR will become more commonly-available over time so there's nothing to stop Jack Black from modifying the School of Rock genome as a class project.
Ed: too farfetched!
Is it now. Think of the Columbia Record Club which sent an LP by U.S. Mail for your approval each month. That was the future in 1965. Now you've got the entire Columbia catalog on your iPhone and you carry it about in your pocket. Projections for the future are almost never radical enough.
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