gene therapy

Development of a novel intranasal oligonucleotide delivery approach for Huntington disease

Huntington disease (HD) is a progressive brain disorder affecting movement, mood, and cognitive skills, caused by inheriting a mutated copy of the huntingtin gene. This results in the production of a mutant huntingtin protein (mHTT) that is toxic to critical nerve cells in the brain. Reducing mHTT using specialized pieces of DNA, called antisense oligonucleotides (ASOs), should slow or prevent disease onset.

Primary Investigator: 
Award Type: 
Year: 
2019

Allele-specific silencing of the mutant huntingtin gene in a mouse model of Huntington disease

Huntington disease is a fatal and inherited neurodegenerative disease. It is characterized by diminished voluntary motor control, cognitive decline and psychiatric disturbance. Symptoms of the disease first appear in the thirties to fifites, with death usually occurring 15 to 20 years later. While there are still no effective therapies for this disease, recent research discoveries have provided insight into how the disease develops. The normal huntingtin gene encodes a protein that is important for neuronal health.

Primary Investigator: 
Award Type: 
Year: 
2011
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