Jennifer Doudna and her colleagues found an enzyme in bacteria that makes editing DNA in animal cells much easier. For this discovery, Jennifer Doudna maybe in line to win a Nobel Prize in the coming decade.
Until now, the tools for fixing or replacing a gene in animals were cumbersome, if they worked at all. CRISPR/Cas9 changes that by allowing scientists to work inside cells, making changes in specific genes much faster and cheaper then ever before. The implications for medicine could be enormous. Let's say two people who are each carriers of the cystic fibrosis gene want to have children but don't want to risk having a child with the disease. Doctors already can use IVF to create an embryo; one day they might also be able to use CRISPR/Cas9 to then fix the damaged gene. It's also possible to imagine treating blood disorders, like sickle cell anemia, that are caused by a single gene.
CRISPR/Cas9 also has huge implication in stem cell gene therapy where in the case of CF, the CFTR gene could be fixed in children and adults with CF in entire organs like the lungs and GI. I reported on this technique here, where CFTR was fixed in the small intestine using cultured intestinal stem cells.
cultured intestinal stem cells
In addition to the positive uses of CRISPR/Cas9, Doudna acknowledges there is a dark side. Genetically modifying human beings brings to mind images of Frankenstein monsters. And the technique could be used for trivial or even harmful uses.
Source: http://t.co/0nrgJkQVfT
- Chris
Until now, the tools for fixing or replacing a gene in animals were cumbersome, if they worked at all. CRISPR/Cas9 changes that by allowing scientists to work inside cells, making changes in specific genes much faster and cheaper then ever before. The implications for medicine could be enormous. Let's say two people who are each carriers of the cystic fibrosis gene want to have children but don't want to risk having a child with the disease. Doctors already can use IVF to create an embryo; one day they might also be able to use CRISPR/Cas9 to then fix the damaged gene. It's also possible to imagine treating blood disorders, like sickle cell anemia, that are caused by a single gene.
CRISPR/Cas9 also has huge implication in stem cell gene therapy where in the case of CF, the CFTR gene could be fixed in children and adults with CF in entire organs like the lungs and GI. I reported on this technique here, where CFTR was fixed in the small intestine using cultured intestinal stem cells.
cultured intestinal stem cells
In addition to the positive uses of CRISPR/Cas9, Doudna acknowledges there is a dark side. Genetically modifying human beings brings to mind images of Frankenstein monsters. And the technique could be used for trivial or even harmful uses.
Source: http://t.co/0nrgJkQVfT
- Chris