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Could CRISPR cure HIV? US biotech Excision raises $60m to find out

pharmaphorum

Once someone is infected with HIV it stays with them for life thanks to the retrovirus’s ability to inject its DNA code into the host. The cash will also help develop two other preclinical drugs, EBT-104 for Herpes Simplex Virus and EBT-107 for hepatitis B.

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Gene editing in organoids: accounting for complexity in drug discovery

Drug Discovery World

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. 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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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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Touchlight boosts DNA manufacturing capacity with latest expansion

Pharmaceutical Technology

Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.

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

Pharmaceutical Technology

On the other hand, a lack of diversity in clinical trial populations, particularly when the disease population is significantly underrepresented, greatly diminishes the quality of data obtained for drug safety and efficacy profiles. This may sound obvious, but it is still a major hurdle in drug development.

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