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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.
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.
The draft guidance recommends that no more than 15 questions are included in the briefing package. CBER will not commit to reviewing packages greater than 250 pages. Where vector presence is detected, transgene mRNA and/or proteinexpression levels should also be measured.
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