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What is a method being explored to silence the huntingtin gene?

User Aetherix
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Final answer:

A method to silence the huntingtin gene involves antisense oligonucleotides (ASOs) which prevent the gene's mRNA from being translated into protein. This approach forms part of a larger set of gene silencing techniques also used in cancer treatment to reverse epigenetic changes and restore normal cell function.

Step-by-step explanation:

One method being explored to silence the huntingtin gene is through the use of antisense oligonucleotides (ASOs). ASOs work by binding to the mRNA produced by the huntingtin gene, blocking its translation into the huntingtin protein, which is implicated in Huntington's disease. This approach is part of a wider array of gene silencing strategies that include RNA interference (RNAi) through siRNAs and miRNAs, as well as epigenetic modifications like DNA methylation and histone deacetylation.

For instance, antisense therapy has been designed to lower the expression of specific genes that are deregulated or overexpressed, resulting in the production of an excess of gene product. By introducing a nucleic acid sequence complementary to the mRNA, antisense oligonucleotides can prevent translation, resulting in a reduced amount of target protein. This therapeutic approach is not only used in genetic diseases but also different types of cancers where it helps to re-establish normal cell growth. Meanwhile, understanding how gene expression in cancer cells differs from that in normal cells can reveal specific targets for drug therapy, enabling the design of drugs that can reverse epigenetic changes and 'switch back on' silenced genes in cancer cells.

User PEREZje
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