Topic > Crispr Technology - The Potential Tool to Treat Huntington's Disease

People with Huntington's disease (HD) are unable to control their movements, lose the ability to think, and have emotional problems. HD is a genetic disease caused by mutations in the Huntingtin gene. DNA segments in the Huntingtin gene repeat abnormally higher 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 Shouldn't Be Banned"? Get an original essay Researchers 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 the “cutting and pasting” of deoxyribonucleic acid (DNA) sequences. Before the CRISPR treatment for HD can be released to the public, extensive research and studies must be done to ensure that CRISPR is safe and effective. DNA is a macromolecule that carries instructions 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 nitrogenous bases: adenine (A), thymine (T), guanine (G), and cytosine (C). DNA instructions and its genetic code are determined by the sequence of these bases. HD is an autosomal dominant disease, meaning that inheriting a single defective huntingtin gene is enough to cause the disease. Every human inherits 2 copies of each gene, one copy is maternal and the other is paternal. A parent with a defective huntingtin gene can pass on both the defective and healthy copies. Therefore, there is a 50% chance of inheriting a defective huntingtin gene from a parent with HD. The faulty Huntingtin gene causes excessive buildup of the Huntingtin protein, which causes damage to nerve cells in some regions of the brain. This causes Huntington's disease and thus hinders neurological function. CRISPR technology uses a short RNA with a base sequence complementary to the desired DNA base sequence. This RNA is 'loaded' into Cas9, which is 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 Cas9 nickase. Instead of cutting both strands of the DNA double helix, nickase made it possible to cut only one of the strands, which allows for much more precise genetic modification. IBC researchers hope to break new ground in neurodegenerative research and quickly move into human trials following the release of their new findings. The IBC research team has developed a new variant of CRISPR using the Cas9 nickase, which is safer and more specific than the previous version. This makes it an “attractive treatment tool for Huntington's disease” as “no sequence-specific side effects were observed” during the testing phase. This new CRISPR variant managed to inactivate the mutant huntingtin gene, ceasing production of the toxic protein. Overall, the researchers were able to edit the huntingtin gene, reducing about 70% of the neurodegenerative proteins. Since not all of this neurodegenerative protein has been eliminated, the team needs to conduct further studies in cellular models of HD to ensure that the.