03 March

Huntington’s disease: P42 peptide Therapy



Huntington’s disease (HD) is an autosomal-dominant, adult onset, neurodegenerative hereditary disorder clinically characterized by the presence of motor dysfunction, cognitive decline, neuropsychiatric symptoms and behavioral problems. In the European Union, the incidence of this rare disease is approximately 1.3 in 10,000 people. Huntington Disease is caused by an expanded CAG repeat in the first exon of the Huntingtin (HTT) gene. Enlarged polyglutamine repetitions of variable lengths containing N-terminus are obtained as a result. These stick together and form intranuclear and intracytoplasmic cellular deposits in the damaged brain cells.

Since years, vigorous study is being carried out to discover promising therapies to eliminate the disease. Current research mainly focuses on the latest development concerning peptide strategy. several therapeutic peptides that significantly slow down the progression of symptoms in experimental models of Huntington’s disease, have been developed.

One of the best approaches is to target very initial steps in the pathophysiological cascade of the disease, such as aggregation or cleavage process. Preventing misfolding and aggregation of the expanded polyQ protein with the help of peptides interacting with polyQ stretches or with Htt protein is a fascinating idea. A 23aa peptide P42, which is a part of the Htt protein, prevents sticking of abnormal Htt protien which therefore prevents consequences of aggregation and improves the symptoms of the disease.