A while back I posted a thread about biotechnology applications which are being used right now to help us, for example to identify strains of bacteria such as the one which caused so much havoc in Germany and other parts of Europe. The work in that case involved some gene sequencing to determine if this was in fact a new serotype, turned out it wasn't.
The biotechnology field is moving so fast...just last week I was at a fish health conference, and heard more about various polymerase chain reactions than about fish physiology.
This week, I bring you news of the next step in managing disease and illness, genome editing. The gist of this new gene therapy is fairly simple in principle, much harder in application. You pinpoint the exact location in the genome where a mutation is found and causes defects, and you repair the error. Soooo much harder in reality. It's not so hard to do with a single strand of DNA in the lab, but in vivo, that is something altogether more complicated.
An article recently published in Nature, detailed how researchers have been able to repair a defect in the livers of lab mice, which causes hemophilia. They did this using an engineered virus. One version of the virus was modified to carry the nuclease enzymes needed to cut the strand, in the exact location of the defect. The other version of the virus delivered the replacement gene to be spliced back into the strand. Truly remarkable stuff.
The nitty gritty of how this was performed, is in the engineered enzyme, a sort of zinc finger nuclease. These enzymes can break the double strand, without unraveling the whole bit, which is what makes this possible inside living cells. The technology needed to efficiently target the exact location where the mutation appears is now available. With this technology, early stage research could provide proof of concept by using a patients own stem cells or available cell lines to repair the defect in vitro, before later phase clinical trials. This is very exciting news!
Source:
http://www.eurekalert.org/pub_releases/2011-06/chop-gea062411.php
The biotechnology field is moving so fast...just last week I was at a fish health conference, and heard more about various polymerase chain reactions than about fish physiology.
This week, I bring you news of the next step in managing disease and illness, genome editing. The gist of this new gene therapy is fairly simple in principle, much harder in application. You pinpoint the exact location in the genome where a mutation is found and causes defects, and you repair the error. Soooo much harder in reality. It's not so hard to do with a single strand of DNA in the lab, but in vivo, that is something altogether more complicated.
An article recently published in Nature, detailed how researchers have been able to repair a defect in the livers of lab mice, which causes hemophilia. They did this using an engineered virus. One version of the virus was modified to carry the nuclease enzymes needed to cut the strand, in the exact location of the defect. The other version of the virus delivered the replacement gene to be spliced back into the strand. Truly remarkable stuff.
The nitty gritty of how this was performed, is in the engineered enzyme, a sort of zinc finger nuclease. These enzymes can break the double strand, without unraveling the whole bit, which is what makes this possible inside living cells. The technology needed to efficiently target the exact location where the mutation appears is now available. With this technology, early stage research could provide proof of concept by using a patients own stem cells or available cell lines to repair the defect in vitro, before later phase clinical trials. This is very exciting news!
Source:
http://www.eurekalert.org/pub_releases/2011-06/chop-gea062411.php
