Huntington's disease and novel genetic therapies

as we know Huntington's disease occurs because of cytosine-adenineguaninerepeats getting expanded in the Huntington's gene resulting in amutant and inefficient huntingtin protein. treatment methods can onlyappease some of the symptoms and this Hereditary doesn't have a certaincure yet. In this article, we are going to Review some promising novelGenetic methods that are being used to either improve these patients' qualityof life or relieve as many symptoms as possible in the earlier stages of theirsickness. People who inherit HD can be identified with genetic tests whethertheir symptoms are observable or not yet present. Recently the most activetherapies that are being studied are in fields, named: DNA/gene, RNAmodulation, and those in which target aberrant downstream pathways. Oneof the recent trials is on mouse models of HD is trying to stop or slow thesevere reduction of brain cholesterol that is mainly produced by astrocytes.This reduction results in neuronal dysfunction in these mouse models and asolution is to transfer the gene that is relevant to transcription factor sterolregulatory element-binding protein ۲ (SREBP۲) and as a result activation ofSREBP۲-controlled genes is lessened. This transfer is done with arecombinant Adeno-associated virus ۲/۵ (AAV۲/۵) which carriestranscriptionally active N-terminal fragment of human SREBP۲ and aimsspecifically at astrocytes. Another trial was done with an Adeno-associatedviral vector containing the gene for a microRNA targeting human HTT,injected to Minipigs.

کلیدواژه ها:

gene therapy ، Huntington ، SREBP۲ ، Adenovirus ، microRNA