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Gene Silencing

Genome

In-Vivo

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo

In-Vivo Gene Editing Trials Drugs

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

Gene Editing

Gene Editing Gene Engineer DNA

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

The use of base editing in stem-cell based therapies

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