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  • Essay / Crispr Technology - The Potential Tool to Cure Huntington's Disease

    People with Huntington's disease (HD) are unable to control their movements, lose their ability to think, and have emotional problems. HD is a genetic disease caused by mutations in the Huntingtin gene. DNA segments of the Huntingtin gene repeat abnormally more often in people with HD. There is currently no cure for HD. This is because the type of mutation varies and the location of the mutation is in different parts of the gene. Say no to plagiarism. Get a tailor-made essay on “Why violent video games should not be banned”?Get the original essayResearchers are testing CRSPR (Clustered Regularly Interspaced Short Palindromic Repeat) technology to develop a treatment for HD. CRISPR is currently the most precise gene editing tool available and involves “cutting and pasting” deoxyribonucleic acid (DNA) sequences. Before CRISPR treatment for HD can be made public, extensive research and studies must be conducted to ensure that CRISPR is safe and effective. DNA is a macromolecule that contains the instructions necessary for the development, reproduction and functioning of an organism. The structure of DNA is a double helix and each strand is made up of nucleotide molecules. DNA is made up of four types of nitrogen bases: adenine (A), thymine (T), guanine (G), and cytosine (C). The instructions of DNA and its genetic code are determined by the sequence of these bases. HD is an autosomal dominant disease, meaning that inheriting just one defective Huntingtin gene is enough to cause the disease. Each human inherits 2 copies of each gene, one copy is maternal and the other is paternal. A parent carrying a defective Huntingtin gene can pass on either the defective copy or the healthy copy. Therefore, there is a 50% chance of inheriting a defective Huntingtin gene from a parent with HD. The faulty Huntingtin gene causes excessive accumulation of the Huntingtin protein, leading to damage to nerve cells in certain areas of the brain. This causes Huntington's disease and therefore impairs neurological function. CRISPR technology uses a short RNA with a base sequence complementary to the desired DNA base sequence. This RNA is “loaded” with Cas9, an enzyme capable of cutting DNA at specific sites. CRSPR technology can be used in human cells and it is possible to use this technology to treat genetic diseases such as HD by modifying faulty genes. Researchers at the Institute of Bioorganic Chemistry (IBC) in Poland used the CRISPR nickase Cas9. Instead of severing both strands of the DNA double helix, nickase was able to sever only one of the strands, allowing for much more precise genetic modification. IBC researchers hope to open new avenues in neurodegenerative research and move quickly to human trials. after the publication of their new findings. The IBC research team developed a new CRISPR variant using Cas9 nickase, which is safer and more specific than the previous version. This makes it an “attractive treatment tool for Huntington’s disease” because “no sequence-specific side effects were observed” during the testing phase. This new CRISPR variant successfully inactivated the mutant huntingtin gene, thereby stopping production of the toxic protein. Overall, the researchers were able to modify the Huntingtin gene, reducing about 70% of neurodegenerative proteins. Like all of this neurodegenerative protein.