A REVIEW ARTICLE ON TREATING HUNTINGTON’S DISEASE: CURRENT AND EMERGING THERAPEUTIC ASPECTS
DOI:
https://doi.org/10.22159/ijms.2025v13i1.53191Keywords:
Huntington Chorea/Huntington’s disease, Genetics, Pathogenesis, Polyglutamine, ZFP, TALENAbstract
A rare genetic neurodegenerative disorder called Huntington’s disease (HD) causes nerve cells to progressively die. When the trinucleotide CAG repeats are <36, it is regarded as “normal.” When the repetitions in the huntingtin gene are >36, polyglutamine (polyQ) tract leads to polyQ poisoning, which in turn causes psychological, genetic, and movement disorders which are hallmarks of HD. One particularly concerning aspect of HD is its controlling inheritance pattern, this indicates that each child born to a parent who exaggerated through the condition has a 50% likelihood of having the lineage of mutated gene that causes the disease. As a result, there is a significant risk that offspring of individuals with HD may also develop the condition. The underlying molecular mechanisms resulting in the visible loss of neurons remain incompletely elucidated, and the current therapeutic approaches primarily aim to alleviate symptoms. Many symptoms can be treated with the HD prescription medications that are currently available. These include prescribed medication for chorea, tranquilizers, calming agents, antidepressants, and non-pharmacological therapy. Additional possible treatments now undergoing clinical research include RNA interference therapies, therapies targeting RNA using tiny molecules, antibody therapies, stem cell therapies, small molecule therapies not targeting RNA, and therapies concentrating on neuroinflammation. Among the potential treatments presently in pre-clinical development stages are zinc-finger protein therapies, transcription activator-like effector nuclease therapies, and rehabilitations involving clustered regularly interspaced short palindromic repeats (CRISPS)/CRISPS-associated system. Therefore, the purpose of this comprehensive review is to discuss the efficacy of current HD medicines and look into the insights of new emerging therapies which are under pre-clinical development stage.
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