RNA interference
RNAi
A technology that was awarded the Nobel prize in 2006, RNAi could be used to target virtually any gene of the human genome that’s causing disease to stop his expression. Thus unlocking molecular targets that were inaccessible before. RNA interference occurs in plants, animals, and humans. It is of great importance for the regulation of gene expression, participates in defense against viral infections, and keeps jumping genes under control. RNA interference is already being widely used in basic science as a method to study the function of genes and it may lead to novel therapies in the future.
Intrigued? Yes, the process for therapeutic development heavily relies on correlating genotype to phenotype. The best way to achieve this is to disrupt gene function and analyze its phenotypic. Researchers can experimentally regulate gene expression and interrogate gene function either at the translational level or at the genetic level using two biological tools.. RNAi and CRISPR. Learn more about CRISPR here..
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Scientists are using the RNAi mechanism to learn more about what particular genes do and how to alter their function.
Determining gene function is a relatively simple matter of inserting double-stranded RNA molecules that have a particular sequence into cells and observing the effects after RNAi silences the corresponding gene. Conceivably, this method may one day be used to silence gene mutations that cause human diseases such as Huntington's disease, rheumatoid arthritis, cancer, and many others. By using either the body's own mutations or viral invaders, scientists may develop a new type of drug—for example, one that switches off the genes of a cancer cell and leaves healthy cells unaffected. However, because RNAi's potential effects are so powerful, scientists must first determine that they can control the mechanism so that only the target gene is silenced, and not others. Macular Degeneration is the leading cause of vision loss, affecting more than 10 million Americans – more than cataracts and glaucoma combined.
At present, Macular Degeneration is considered an incurable eye disease. Macular Degeneration is caused by the deterioration of the central portion of the retina, the inside back layer of the eye that records the images we see and sends them via the optic nerve from the eye to the brain. The retina’s central portion, known as the macula, is responsible for focusing central vision in the eye, and it controls our ability to read, drive a car, recognize faces or colors, and see objects in fine detail. |
1 of 4: the discovery of rna interface
2 of 4: RNAi: Slicing, dicing and serving your cells
3 of 4: RNAi, a Promising Potential New Class of Medicines
4 of 4: an attempt to manipulate gene expression.
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- Transcription factors: are proteins that regulate the transcription of genes that is, their copying into RNA, on the way to making a protein.
- Transcription: is the process where a gene's DNA sequence is copied (transcribed) into an RNA molecule. Transcription is a key step in using information from a gene to make a protein.
- Gene expression: is when a gene in DNA is "turned on," that is, used to make the protein it specifies. Not all the genes in your body are turned on at the same time, or in the same cells or parts of the body.
- What has to happen for a gene to be transcribed? The enzyme RNA polymerase, which makes a new RNA molecule from a DNA template, must attach to the DNA of the gene. It attaches at a spot called the promoter.