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. Brain-wide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editor. Online June 27, 2024.

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Disease Therapies Clinical Trials Engineering 142

Drug Target Review

SEPTEMBER 10, 2024

For example, advances in media and feeds have contributed to higher cell protein expression by optimising nutrient availability and reducing accumulation of waste products within a culture. Novel vector design and synthetic promoters have been designed to maximise productivity and enhance gene expression stability.

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FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. Where vector presence is detected, transgene mRNA and/or protein expression levels should also be measured. Tobolowsky & Charles G. Raver & James E.

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Therapies FDA Production Disease 59