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RNA interference method could treat muscular dystrophy

Drug Discovery World

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.

RNA 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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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