In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference10 articles.
1. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis;Gillmore;N. Engl. J. Med.,2021
2. The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug;Johnson;J. Mol. Biol.,2012
3. Transthyretin: roles in the nervous system beyond thyroxine and retinol transport;Oliveira;Expert Rev. Endocrinol. Metab.,2012
4. Tetramer dissociation and monomer partial unfolding precedes protofibril formation in amyloidogenic transthyretin variants;Quintas;J. Biol. Chem.,2001
5. Safety and efficacy of RNAi therapy for transthyretin amyloidosis;Coelho;N. Engl. J. Med.,2013
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