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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

Taking place at the ExCel in London from 4-5 October, ELRIG Drug Discovery 2022 is set to provide visitors with access to the latest trends, technologies and expertise driving the life sciences sector. . Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’.

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2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

It has been a year dominated by the pandemic and many life sciences research projects were put on hold as big pharma turned its attention to vaccines and therapies. But there was some considerable progress in other fields of medicine even though research efforts were diverted away, reports Richard Staines.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself.

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UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data.

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Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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