CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Abstract

In a small group of patients with hereditary ATTR amyloidosis with polyneuropathy, administration of NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of <i>TTR</i>. (Funded by Intellia Therapeutics and Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT04601051.).

Keywords

TransthyretinCRISPRGenome editingCas9AmyloidosisGeneIn vivoEndonucleaseBiologyGenetic enhancementComputational biologyMolecular biologyBiochemistryGeneticsMedicinePathology

MeSH Terms

Amyloid NeuropathiesFamilialCRISPR-Cas SystemsFemaleGene EditingGene Transfer TechniquesHumansInfusionsIntravenousLiposomesMaleMiddle AgedNanoparticlesPrealbuminRNAGuideCRISPR-Cas SystemsRNAMessenger

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Publication Info

Year: 2021
Type: article
Volume: 385
Issue: 6
Pages: 493-502
Citations: 1432
Access: Closed

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APA Style

                            
                                
                                    Julian D. Gillmore, 
                                
                                    Ed Gane, 
                                
                                    Jörg Täubel
                                
                                et al.
                            
                            (2021). 
                            CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. 
                            New England Journal of Medicine
                            , 385
                            (6)
                            , 493-502.
                            https://doi.org/10.1056/nejmoa2107454
                        

Identifiers

DOI: 10.1056/nejmoa2107454
PMID: 34215024

Data Quality

Data completeness: 90%