Huntington’s Disease and Gene Therapy

Huntington's Disease and Gene Therapy Huntington's Disease (HD) is an autosomal dominant neurodegenerative disease that causes a mutation in the Huntingtin protein (Htt) protein (1) (Zuccato et al., 2010). HD is a glutamine-encoding trinucleotide sequence CAG clinically showing progressive chorea, dementia and psychiatric disorder (2) (Leegwater-Kim and Cha, 2004) and planning and memory impairment (3) (Mrzljak and Munoz) Repeat. - San Juan, 2013). Pathologically, HD includes widespread degeneration of moderate spiny neurons (4) (Benraiss and Goldman, 2011) and reactive gliosis (2), in which astrocytes are involved during injury or disease Change to (5) (soft)

Recent advances in gene therapy have enabled new treatments for neurodegenerative diseases such as Parkinson's disease and Huntington's disease and they have provided exciting therapeutic results in appropriate animal models of the corresponding human disease. Phase I clinical trials of these neurodegenerative diseases are scheduled to be initiated or to be initiated.

Huntington's disease is a progressive neurodegenerative disease, a well-known example of autosomal dominant monogenic disease. Most people with single-copy mutant huntingtin gene (HTT) will have Huntington's disease in later years. In many cases, autosomal dominant diseases affect their earliest individuals and prevent them from passing through infancy or childhood, which in turn causes these individuals to multiply and perhaps suddenly in their descendants It prevents passing the mutation. However, in the case of Huntington's disease, the late onset signifies that many affected people already had children before they even noticed that they had a mutation.

Some of the debilitating person's diseases like Huntington's disease are caused by mutations in one of about 20,000 of your genes. Huntington's disease is a fatal progressive neurodegenerative disease, but despite knowing the causative gene, scientists have not yet found a cure or an effective treatment. Huntington's disease is caused by repetitive amplification of a gene called Huntingtin protein. Genes whose defects have prolonged produce proteins that are similar to those that are thin and defective, and they accumulate in the brain. These huntingtin protein masses ultimately result in uncontrolled spasmatic movement that characterizes the disease, as well as various cognitive and psychiatric symptoms.