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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

RNA 40
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The use of base editing in stem-cell based therapies

Drug Discovery World

A second generation CRISPR-Cas gene editing system, base editing, reduces the risk of potentially deleterious effects associated with other gene editing technologies, presenting a safe and effective means of editing cells for therapeutic applications. 2020;13:dmm042317. doi:10.1242/dmm.042317. 13: Cuomo A, Seaton D, McCarthy D, et al.