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Tweet New way of genome editing could cure hemophilia in mice; may be safer than older method, study shows A technique smosh games alliance editing services by Stanford researchers could provide a safer, longer-lasting method of replacing faulty, disease-causing genes with working copies.
Now, researchers at the Stanford University School of Medicine have devised a new way to carry out this genetic sleight of hand. These differences may make the new approach both safer and longer-lasting.
Using the technique, the Stanford researchers were able to cure mice with hemophilia by inserting a gene for a clotting factor missing in the animals.
Although the insertion was accomplished in only about 1 percent of liver cells, those cells made enough of the missing clotting factor to ameliorate the disorder.
Kay is the senior author of the research, published Oct. The lead author is postdoctoral scholar Adi BarzelPhD. When that virus comes around again, the cell uses the snippets of saved genetic material to identify and latch onto matching regions in the viral genome.
Once attached, it cuts the viral genes at precise locations with a protein called Cas9. This allows the rapid creation of genetically modified laboratory animals; what used to take months or years can now take days or weeks.
Site-specific gene targeting is one of the fastest growing fields in gene therapy and genome engineering. Researchers are concerned that, if used in humans, Cas9 may cut the DNA at unexpected locations, which could disrupt or kill the cell.
Alternatively, the promoter of the new gene could adversely affect the expression of nearby genes, causing cancers or other diseases. The foreign bacterial proteins could also cause an immune reaction in patients. I wanted to come up with a novel gene-targeting scheme that involved no vector-borne promoter and did not require the use of an endonuclease.
Instead, the researchers hitch the expression of the new gene to that of a highly expressed gene in the liver called albumin.
The albumin gene makes the albumin protein, which is the most abundant protein in blood.
The researchers used a modified version of a virus commonly used in gene therapy called adeno-associated virus, or AAV.
In the modified version, called a viral vector, all viral genes are removed and only the therapeutic genes remain. They also relied on a biological phenomenon known as homologous recombination to insert the clotting factor gene near the albumin gene.
By using a special DNA linker between the genes, the researchers were able to ensure that the clotting factor protein was made hand-in-hand with the highly expressed albumin protein. During homologous recombination, which is a natural repair process, the cell takes advantage of the fact that it has two copies of every chromosome.Search for your organization from the list below.
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In October , Retro Studios was founded as an alliance between Nintendo and former Iguana Entertainment founder Jeff Spangenberg. Nintendo saw an opportunity for the new studio to create games for the upcoming GameCube targeting an older demographic, in the same vein as Iguana Entertainment's successful Turok series for the Nintendo The Hollywood Post Alliance also honored Ray Dolby with its Charles S.
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