hhmi Biolnteractive Central Dogma and Genetic Medicine 3. Mutations in the DNA c
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hhmi Biolnteractive Central Dogma and Genetic Medicine 3. Mutations in the DNA can affect the structure and function of proteins. Some mutations may even cause Click& Learn Student Worksheet genetic diseases. Scientists and doctors can intervene at different points during gene expression to develop treatments for such genetic diseases-or genetic medicine. Let's learn about the genetic medicines that are being developed Select the "Genetic Medicine" tab located on the top right of the screen. Click on the tab corresponding to the genetic medicine(s) that your instructor assigns to you, or scroll through the interactive and click on the pink "+" sign labeled with that genetic medicine. Read the "Genetic Medicine" tab material, watch the video, and read the information in the "Learn more" link. Then, complete the appropriate row(s) below Short Summary Write a one-sentence summary of how this genetic medicine works. Detailed Description Describe how this genetic medicine would be used to treat a genetic disease. (For example, mention how it would fix the disease-causing mutation and/or result in a functioning protein.) Genetic CRISPR Cas9 Gene Therapy Gene Switches Exon Skipping RNA Interference Small Molecule Drug Genetics www.Biolnteractive.org Published January 2018 Page 2 of 4Explanation / Answer
Genetic Medicine Short Summary Detailed Description CRISPR-Cas9 CRISPR-Cas9 is a technology that allows to change or edit a defective gene or DNA of a cell at a precise location. CRISPR-Cas9 comprises a nuclease called Cas9, paired with a guide RNA which bind to a specific DNA sequence in the cell. It guides Cas9 to that target sequence, where the enzyme cleaves the DNA, leaving a double-stranded, blunt-end break. Once the DNA is cut, the cell's DNA repair machinery used to replace an existing segment with a DNA sequence they designed. Gene Therapy Gene therapy is an experimental technology that provides functioning copies of genes to cells with disease-causing versions of those genes. In this the viral vectors with the therapeutic gene. When a viral vector is introduced into the body, it infects target cells and releases its genome, including the therapeutic gene, inside. The cell's machinery then produces a functional protein from the introduced gene. Gene Switches This technology targets the noncoding sequence or regulatory sequence to control the gene expression By regulating the noncoding sequence or regulatory sequence we can control the gene expression. By controlling gene expression we can use it either to prevent genes linked to diseases from being turned on or to keep beneficial genes from being turned off. Exon Skipping Exon skipping is a technology that changes how the primary RNA transcript of a gene with a disease-causing mutation is spliced, removing the mutation from the resulting mRNA. In this technology a specific antisense RNA is introduced in a patient's cells which it binds to the target sequence in the primary RNA transcript, causing the splicing machinery to "skip over" a segment of the transcript. The one or more exons may be spliced out of the resulting mature mRNA, including the exon with the disease-causing mutation. RNA Interference RNA interference (RNAi) technologies involve small RNA segments that target various mRNAs for destruction, reducing the expression of certain genes. In this small RNA segments are inserted into the cell which hybridized with the complementary mRNA, then cell defense machinery degrades the ds RNA and does not allow the expression of genes. Small Molecule Drug Small-molecule drug includes group of chemical compounds of low molecular weight. chemical compounds with low molecular weight can easily absorb by the cell as well as they easily binds to the target site or receptors to show their activity.
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