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Huntington’s Disease: Understanding Its Novel Drugs and Treatments
An inherited neurodegenerative ailment called Huntington's disease (HD) of gradual physical impairment, cognitive decline, and psychiatric symptoms. It is brought on by a mutation of the HTT gene, which causes aberrant huntingtin protein buildup in neurons. This predominantly affects the striat...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Cureus
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10664735/ https://www.ncbi.nlm.nih.gov/pubmed/38021751 http://dx.doi.org/10.7759/cureus.47526 |
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author | Dhingra, Hitaansh Gaidhane, Shilpa A |
author_facet | Dhingra, Hitaansh Gaidhane, Shilpa A |
author_sort | Dhingra, Hitaansh |
collection | PubMed |
description | An inherited neurodegenerative ailment called Huntington's disease (HD) of gradual physical impairment, cognitive decline, and psychiatric symptoms. It is brought on by a mutation of the HTT gene, which causes aberrant huntingtin protein buildup in neurons. This predominantly affects the striatum and cerebral cortex, where neuronal malfunction and eventual cell death follow. The quality index of life for both patients and their families is significantly impacted when symptoms first appear in mid-adulthood. An overview of the available therapies for HD is given in this article. Although HD has no known treatment options, there are several that try to lessen symptoms and reduce the disease's development. By lowering involuntary movements, pharmaceutical treatments like tetrabenazine and deutetrabenazine focus on motor symptoms. Antidepressants and antipsychotic medicines are also used to manage the mental and cognitive symptoms of HD. The investigation of prospective gene-based medicines is a result of research into disease-modifying medications. Reduced synthesis of mutant huntingtin protein is the goal of RNA interference (RNAi) strategies, which may halt the course of illness. Additionally, continuing research into Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9 (CRISPR-Cas9) and other gene editing methods shows promise for reversing the genetic mutation that causes HD. Individuals with HD can benefit from non-pharmacological therapies such as physical therapy, speech therapy, and occupational therapy to increase their functional abilities and general well-being. Supportive treatment, psychiatric therapy, and caregiver support groups are also essential in addressing the difficult problems the illness presents. In conclusion, tremendous progress is being made in the domain of HD treatment, with an emphasis on symptom control, disease modification, and prospective gene-based therapeutics. Even though there has been significant improvement, more study is still required to provide better therapies and ultimately discover a solution for this debilitating condition. |
format | Online Article Text |
id | pubmed-10664735 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Cureus |
record_format | MEDLINE/PubMed |
spelling | pubmed-106647352023-10-23 Huntington’s Disease: Understanding Its Novel Drugs and Treatments Dhingra, Hitaansh Gaidhane, Shilpa A Cureus Preventive Medicine An inherited neurodegenerative ailment called Huntington's disease (HD) of gradual physical impairment, cognitive decline, and psychiatric symptoms. It is brought on by a mutation of the HTT gene, which causes aberrant huntingtin protein buildup in neurons. This predominantly affects the striatum and cerebral cortex, where neuronal malfunction and eventual cell death follow. The quality index of life for both patients and their families is significantly impacted when symptoms first appear in mid-adulthood. An overview of the available therapies for HD is given in this article. Although HD has no known treatment options, there are several that try to lessen symptoms and reduce the disease's development. By lowering involuntary movements, pharmaceutical treatments like tetrabenazine and deutetrabenazine focus on motor symptoms. Antidepressants and antipsychotic medicines are also used to manage the mental and cognitive symptoms of HD. The investigation of prospective gene-based medicines is a result of research into disease-modifying medications. Reduced synthesis of mutant huntingtin protein is the goal of RNA interference (RNAi) strategies, which may halt the course of illness. Additionally, continuing research into Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9 (CRISPR-Cas9) and other gene editing methods shows promise for reversing the genetic mutation that causes HD. Individuals with HD can benefit from non-pharmacological therapies such as physical therapy, speech therapy, and occupational therapy to increase their functional abilities and general well-being. Supportive treatment, psychiatric therapy, and caregiver support groups are also essential in addressing the difficult problems the illness presents. In conclusion, tremendous progress is being made in the domain of HD treatment, with an emphasis on symptom control, disease modification, and prospective gene-based therapeutics. Even though there has been significant improvement, more study is still required to provide better therapies and ultimately discover a solution for this debilitating condition. Cureus 2023-10-23 /pmc/articles/PMC10664735/ /pubmed/38021751 http://dx.doi.org/10.7759/cureus.47526 Text en Copyright © 2023, Dhingra et al. https://creativecommons.org/licenses/by/3.0/This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. |
spellingShingle | Preventive Medicine Dhingra, Hitaansh Gaidhane, Shilpa A Huntington’s Disease: Understanding Its Novel Drugs and Treatments |
title | Huntington’s Disease: Understanding Its Novel Drugs and Treatments |
title_full | Huntington’s Disease: Understanding Its Novel Drugs and Treatments |
title_fullStr | Huntington’s Disease: Understanding Its Novel Drugs and Treatments |
title_full_unstemmed | Huntington’s Disease: Understanding Its Novel Drugs and Treatments |
title_short | Huntington’s Disease: Understanding Its Novel Drugs and Treatments |
title_sort | huntington’s disease: understanding its novel drugs and treatments |
topic | Preventive Medicine |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10664735/ https://www.ncbi.nlm.nih.gov/pubmed/38021751 http://dx.doi.org/10.7759/cureus.47526 |
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