Epigenetics Research: Open Access
Open Access

Short Note on Gene Transfer in the Experimental Model of Huntington's Disease

Bogna Zborowska^{* *}Correspondence: Bogna Zborowska, Department of Epigenetic Research, Bankstown Hospital, Bankstown, Australia, Email:

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Description

Huntington's Disease (HD) is a genetic neurodegenerative disorder characterized by the progressive loss of progressive movement, cognitive decline, and psychiatric symptoms. It is caused by a mutation in the Huntingtin (HTT) gene, which leads to the production of a toxic mutant protein. Researchers and scientists have been exploring various strategies to better understand the disease and develop potential treatments. One of these strategies involves gene transfer in experimental models of Huntington's Disease. In this article, we will provide a short overview of the use of gene transfer techniques in HD research.

Huntington's Disease is a genetic disorder that is inherited in an autosomal dominant manner. This means that if one of our parents has the mutated HTT gene, we have a 50% chance of inheriting the mutation, and if we, we will develop the disease. The mutation involves the expansion of a specific sequence of DNA, known as CAG repeats, in the HTT gene. The greater the number of CAG repeats, the earlier the onset of HD symptoms. The mutant HTT protein leads to the formation of toxic aggregates within neurons, disrupting their normal functions and causing cell death. Research into HD has primarily focused on understanding the underlying molecular mechanisms and finding ways to either slow down or halt the progression of the disease.

Under the arrangement of focal doctrine, the DNA hereditary code decides the RNA that will be deciphered in supplement to the DNA; then, this RNA will proceed to be converted into a protein that has a specific capability in cells. The CAG present in the quality will bring the deciphered protein Huntingtin (HTT) containing long stretches of glutamine that make it to misfolding and ultimately cause the accumulation of the protein, at that point, loses its unique capability. This freak protein causes clinical side effects like chorea, dystonia, incoordination, mental deterioration, and social challenges.

Most neurodegenerative issues are not yet completely comprehended as far as their hereditary bases. In any case, since the essential role of HD is hereditary objective, that would link to the Htt quality and decrease its behaviour. Numerous medicines that were directed for HD planned to focus on this particular quality in various types of quality treatment. One of the techniques utilized for quality treatment was the revelation of RNA impedance. Thus Quality treatment helps at the posttranscriptional level. Viral vectors are normally utilized as the conveyance strategy, particularly AAV vectors. It was effective in conveying the RNA to mice and added to stifling freak Htt conglomeration development.

Conclusion

Gene transfer techniques have emerged as a determined way for understanding and potentially treating Huntington's Disease. While there is a lot of work to be done, the progress in experimental models of HD offers hope for the development of effective therapies that could significantly impact the lives of those affected by this devastating neurodegenerative disorder. Continued research, careful clinical trials, and ethical considerations will be crucial in realizing the potential of gene transfer techniques to find a cure against Huntington's Disease. Gene transfer techniques have allowed scientists to target and suppress the expression of the mutant HTT gene, which is a crucial step in slowing down the progression of the disease.