Molecular Therapy — Methods & Clinical Development · 2022 · 11 citations · 41 references
Life SciencesGeneticsMolecular GeneticsGenetic EngineeringGenome EditingMedical GeneticsAllele-specific EditingCrispr-based EngineeringCrispr-cas SystemsGenetic DisordersTherapeutic Genome EditingAllele-specific Genome EditingAllele-specific KnockoutAllele-specific TherapyCrispr-cas TherapeuticsHematopoietic Stem Cell EditingIn Vivo Genome EditingMutant Allele DisruptionTherapeutic Allele Silencing
knockout was specific to the mutant allele and involved no off-targets. These results demonstrate the therapeutic potential of selective allele editing that may be applicable to SCN and other autosomal dominant disorders.
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Killing activity of neutrophils is mediated through activation of proteases by K+ flux
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Mutations in the gene encoding neutrophil elastase in congenital and cyclic neutropenia
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