Amanda Carotenuto
http://www.hcplive.com/publications/hemophilia-reports/2014/december2014/Hemophilia-Cured-in-Mice-Models-Using-New-Genome-Editing-Method-
Rachek Lutz
5 January 2015
Summary: A new genome editing method has been developed by researchers at Stanford University School of Medicine. This technique has successfully cured hemophilia in mice, and could possibly lead to longer lasting and safer methods then the one that currently exists. This was accomplished by inserting a clotting factor gene into about 1% of the mouse's liver cells, which produced enough of those cells with the added gene to cure this disease. It is believed that this method opens up the possibility of curing genetic disease for entire lifetimes. This new method also does not use nucleases and promoters to cut up and activate the gene that has been inserted, and is safer to use, as promoters and nucleases may have "significant adverse effects".
Connection: This study connects to the unit on molecular genetics, as well as the one on heredity. In the molecular genetics unit, we learned about nucleases and how genes are turned on and off. The unit also covered genetic engineering, and how genes are cut up and moved or replaced, which is what the above method is attempting to replace. It also does not need promoters, which we learned about how it would be where the RNA polymerase would attach in order to activate the inserted gene. However the above method does not require this or the restriction enzymes, making it safer then the original method.
Summary: A new genome editing method has been developed by researchers at Stanford University School of Medicine. This technique has successfully cured hemophilia in mice, and could possibly lead to longer lasting and safer methods then the one that currently exists. This was accomplished by inserting a clotting factor gene into about 1% of the mouse's liver cells, which produced enough of those cells with the added gene to cure this disease. It is believed that this method opens up the possibility of curing genetic disease for entire lifetimes. This new method also does not use nucleases and promoters to cut up and activate the gene that has been inserted, and is safer to use, as promoters and nucleases may have "significant adverse effects".
Connection: This study connects to the unit on molecular genetics, as well as the one on heredity. In the molecular genetics unit, we learned about nucleases and how genes are turned on and off. The unit also covered genetic engineering, and how genes are cut up and moved or replaced, which is what the above method is attempting to replace. It also does not need promoters, which we learned about how it would be where the RNA polymerase would attach in order to activate the inserted gene. However the above method does not require this or the restriction enzymes, making it safer then the original method.
It's a great discovery they discovered this editing method to work in mice, but how can we guarantee that it will continue to work in humans or larger animals as the genomes differ greatly?
ReplyDeleteSimilarly to Anna's question, can this technique be used to cure hemophilia in other species? If so how successful would it be?
ReplyDeleteWhat causes the adeno-associated virus to have longer lasting effects on mice than on humans?
ReplyDelete