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In the first demonstration of gene therapy's potential for tackling blindness, three teams of researchers have used the technique to successfully treat people with Leber congenital amaurosis (LCA).
For demonstration of gene expression changes, the mean fold changes as obtained from the ΔΔCT-method and their standard deviations were calculated.
However, a functional demonstration was lacking, the time-course of activation was not investigated and an independent demonstration of gene expression was not available.
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This is the first demonstration of gene-based restoration of both visual function/vision-elicited behavior and cone preservation in a mammalian model of GC1 deficiency.
All previous demonstrations of gene therapy in animal models of SS have utilized a virus as the gene transfer vector.
Recent demonstrations of gene transfer and recombination among chlamydiae temper the correlation between serotype and AAX functionality [ 13].
A more complete understanding of the genetic basis of pathogenicity could provide targets for gene regulation, leading to methods of disease control The recent demonstration of targeted gene disruption in O. novo-ulmi by RNA interference [ 20], combined with knowledge about specific target genes as detected by EST analysis, makes this goal more readily achievable.
The demonstration of distinct gene sets with preference for a certain brain region suggests that the annotations of these genes might reflect functional specialisations of the given regions.
From that came the first demonstration of a gene deletion.
Notable achievements are the first demonstration of localized gene editing in (mouse) epidermis and a pilot demonstration of localized efficacy of a therapeutic siRNA in a human who has a keratin mutation.
Collectively, these data provided the first demonstration of a gene knock down in the chinchilla.
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