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| Gene targeting raises cure hopes | Gene targeting raises cure hopes |
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| A more efficient way to shut down rogue genes raises hopes of new therapies for conditions like diabetes and HIV. | A more efficient way to shut down rogue genes raises hopes of new therapies for conditions like diabetes and HIV. |
| Systematically knocking out single genes potentially gives scientists unprecedented control over the processes which cause disease. | Systematically knocking out single genes potentially gives scientists unprecedented control over the processes which cause disease. |
| US and UK researchers have developed synthetic proteins which can target individual genes quickly, simply and with a high degree of success. | US and UK researchers have developed synthetic proteins which can target individual genes quickly, simply and with a high degree of success. |
| The study appears in Proceedings of the National Academy of Sciences. | The study appears in Proceedings of the National Academy of Sciences. |
| The new technique is already being used in trials on a range of diseases. | The new technique is already being used in trials on a range of diseases. |
| The single step process is extremely quick and reliable and opens up exciting possibilities for research and gene therapy Sir Aaron KlugMRC Laboratory of Molecular Biology in Cambridge | The single step process is extremely quick and reliable and opens up exciting possibilities for research and gene therapy Sir Aaron KlugMRC Laboratory of Molecular Biology in Cambridge |
| The regulation of gene expression is controlled by regulatory proteins called transcription factors. | The regulation of gene expression is controlled by regulatory proteins called transcription factors. |
| The researchers have created their own synthetic versions of these transcription factors called zinc-finger nucleases. | The researchers have created their own synthetic versions of these transcription factors called zinc-finger nucleases. |
| They can be used to cut into and inactivate a target gene in a very precise way, without affecting other nearby genes. | They can be used to cut into and inactivate a target gene in a very precise way, without affecting other nearby genes. |
| It is also possible to use a similar approach to activate beneficial genes by introducing new DNA at the point where the gene is cut. | |
| The research initiated by Sir Aaron Klug, based at the Medical Research Council's Laboratory of Molecular Biology in Cambridge. His work has been taken on by US firm Sangamo BioSciences. | The research initiated by Sir Aaron Klug, based at the Medical Research Council's Laboratory of Molecular Biology in Cambridge. His work has been taken on by US firm Sangamo BioSciences. |
| Precision targeting | Precision targeting |
| The targetting of individual genes is currently carried out using a process known as homologous recombination, which involves the exchange of genetic material between the target gene and DNA created in the lab. | The targetting of individual genes is currently carried out using a process known as homologous recombination, which involves the exchange of genetic material between the target gene and DNA created in the lab. |
| However, this process is time consuming, can be very elaborate, requiring the insertion of markers into the target gene, and is not always very efficient. | However, this process is time consuming, can be very elaborate, requiring the insertion of markers into the target gene, and is not always very efficient. |
| Sir Aaron said: "The beauty of zinc-finger nucleases lies in their simplicity. | Sir Aaron said: "The beauty of zinc-finger nucleases lies in their simplicity. |
| "Where other methods are long, arduous and often messy, it is relatively easy to switch off genes using this method. | "Where other methods are long, arduous and often messy, it is relatively easy to switch off genes using this method. |
| "The zinc-finger design allows us to target a single gene, while the nuclease disrupts the gene. | "The zinc-finger design allows us to target a single gene, while the nuclease disrupts the gene. |
| "The single step process is extremely quick and reliable and opens up exciting possibilities for research and gene therapy." | "The single step process is extremely quick and reliable and opens up exciting possibilities for research and gene therapy." |
| Animal trials are already under way to use the technique to knock out the receptor of HIV in immune system T-cells of patients with Aids. | Animal trials are already under way to use the technique to knock out the receptor of HIV in immune system T-cells of patients with Aids. |
| If successful this will render the T-cells immune from HIV infection, and enable them to fight disease. | If successful this will render the T-cells immune from HIV infection, and enable them to fight disease. |
| Clinical trials to aid patients with problem blood vessels are also under way. | Clinical trials to aid patients with problem blood vessels are also under way. |
| Dr Vincent Cunliffe, a genetics expert from the University of Sheffield, described the technique as "a powerful method for efficiently and selectively inactivating" individual genes. | Dr Vincent Cunliffe, a genetics expert from the University of Sheffield, described the technique as "a powerful method for efficiently and selectively inactivating" individual genes. |
| He said: "This technology will have many applications in future research." | He said: "This technology will have many applications in future research." |