Litcius/Paper detail

Prospects for gene replacement therapies in amyotrophic lateral sclerosis

Ilaria Giovannelli, Adrian Higginbottom, Janine Kirby, Mimoun Azzouz, Pamela J. Shaw

2022Nature Reviews Neurology22 citationsDOI

Topics & Concepts

Amyotrophic lateral sclerosisRiluzoleMedicineDiseaseSpinal muscular atrophyLoss functionGenetic enhancementSMN1NeuroscienceBioinformaticsGenePathologyBiologyGeneticsPhenotypeNeurogenetic and Muscular Disorders ResearchAmyotrophic Lateral Sclerosis ResearchAutism Spectrum Disorder Research