Article
A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction.
Gene therapy - 1 Sept 1996
Dorin J R, Farley R, Webb S, Smith S N, Farini E, Delaney S J, Wainwright B J, Alton E W, Porteous D J
Abstract excerpt
Quantifying the level of transgene expression necessary for phenotypic effect is an important consideration in designing somatic gene therapy protocols. A nonlinear relationship between phenotype and gene activity is predicted by control analysis for any autosomal recessive condition. The unaffected phenotype of heterozygotes for autosomal recessive disorders demonstrates that 50% of the normal level of gene...
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