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Gene editing in organoids: accounting for complexity in drug discovery

Drug Discovery World

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . What are organoids? .

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RNA interference method could treat muscular dystrophy

Drug Discovery World

One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. Using an experimental technique called exon skipping by antisense oligonucleotides, the team corrected a mistake in the FKTN gene that blocks the chemical glycosylation of a biologically important protein. . Modifying RNA function.

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ELRIG meeting will focus on genome engineering in drug discovery

Drug Discovery World

The meeting will focus on the application of genome engineering in identification of novel drug targets through large-scale CRISPR based functional genomics studies, developing advanced cellular and in vivo disease models for target validation, and its applications in therapeutic genome editing. Register here.

Genome 52
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The use of base editing in stem-cell based therapies

Drug Discovery World

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.

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Clinical Catch-Up: January 18-22 | BioSpace

The Pharma Data

Brilacidin has shown in vitro to be have antiviral properties against different SARS-CoV-2 strains and other human coronaviruses. With a presidential inauguration and a federal holiday, it wasn’t an enormously busy week for clinical trial news, but there was a fair amount, nonetheless. Read on to see. COVID-19-Related. Non-COVID-19-Related.

Trials 52
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Meet the company on a mission to transform research and advance healthcare

Pharmaceutical Technology

He says these issues must be urgently addressed over the next few years as advancing medical knowledge and the role of genomics in treating disease ushers in a new era of precision medicine and new potential to address diseases that affect certain populations. This may sound obvious, but it is still a major hurdle in drug development.

Research 130