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CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Alan Solomon

2021New England Journal of Medicine37 citationsDOIOpen Access PDF

Topics & Concepts

CRISPRGenome editingMedicineTransthyretinAmyloidosisGeneComputational biologyIn vivoGeneticsBioinformaticsPathologyBiologyAmyloidosis: Diagnosis, Treatment, OutcomesRenal Diseases and GlomerulopathiesCellular transport and secretion
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