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Remove 2024 Remove Animal Testing Remove Packaging Remove Protein Expression
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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

JUNE 27, 2024

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. The first problem they had to tackle was the editor’s size, because the editor needs to be small enough to be packaged and delivered to specific cells in the body.

Disease 142
Disease Therapies Engineering Clinical Trials 142
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Rapid delivery of toxicological material

Drug Target Review

SEPTEMBER 10, 2024

However, developing an accelerated IND application requires that good laboratory practice (GLP) toxicological data must be generated, collected, interpreted and integrated in the IND-enabling data package. Novel vector design and synthetic promoters have been designed to maximise productivity and enhance gene expression stability.

Clinical Supply 64
Clinical Supply Drug Development Production Animal Testing 64
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