Article
Strategems in vitro for gene therapies directed to dominant mutations.
Human molecular genetics - 1 Sept 1997
Millington-Ward S, O'Neill B, Tuohy G, Al-Jandal N, Kiang A S, Kenna P F, Palfi A, Hayden P, Mansergh F, Kennan A, Humphries P, Farrar G J
Abstract excerpt
A major difficulty associated with the design of gene therapies for autosomal dominant diseases is the immense intragenic heterogeneity often encountered in such conditions. In order to overcome such difficulties we have designed, and evaluated in vitro, three strategies which avoid a requirement to target individual mutations for genetic suppression. In the first, normal and mutant alleles are suppressed by...
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