Article
Toward a gene therapy for dominant disease: validation of an RNA interference-based mutation-independent approach.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2005
Kiang Anna-Sophia, Palfi Arpad, Ader Marius, Kenna Paul F, Millington-Ward Sophia, Clark Gerry, Kennan Avril, O'reilly Mary, Tam Lawrence C T, Aherne Aileen, McNally Niamh, Humphries Pete, Farrar G Jane
Abstract excerpt
The intragenic heterogeneity encountered in many dominant disease-causing genes represents a significant challenge with respect to development of economically viable therapeutics. For example, 25% of autosomal dominant retinitis pigmentosa is caused by over 100 different mutations within the gene encoding rhodopsin, each of which could require a unique gene therapy. We describe here an RNA interference...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.