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AAV-based in vivo gene therapy for neurological disorders

Qinglan Ling, Jessica A. Herstine, Allison M. Bradbury, Steven J. Gray

2023Nature Reviews Drug Discovery187 citationsDOI

Topics & Concepts

Genetic enhancementAdeno-associated virusMedicineTransgeneClinical trialDiseaseVector (molecular biology)BioinformaticsGene deliveryAdverse effectDrug developmentNeuroscienceDrugPharmacologyGeneBiologyPathologyGeneticsRecombinant DNAVirus-based gene therapy researchRNA Interference and Gene DeliveryCRISPR and Genetic Engineering
AAV-based in vivo gene therapy for neurological disorders | Litcius