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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 genesilencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).
The tool could also prove to be safer than conventional CRISPR-based genetherapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenetic Editing with CRISPR.
those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo genetherapies, and 2 gene-modified cell therapies. greater safety, transient and reversible effect on gene expression).
The tool could also prove to be safer than conventional CRISPR-based genetherapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenome Editing with CRISPR.
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