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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. The genetic anomaly blocks the chemical glycosylation of a biologically important protein. Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. .

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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. It includes adults with confirmed COVID-19 on supplemental oxygen or mechanical ventilation with abnormal levels of at least two serum biomarkers, C reactive protein, D dimer, lactate dehydrogenase, or ferritin.

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