Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing
Jakob M. Domm, Sarah K. Wootton, Jeffrey A. Medin, Michael L. West
Topics & Concepts
Genetic enhancementGenome editingGene deliveryFabry diseaseBiologyDiseaseViral vectorGeneBioinformaticsMedicineCRISPRGeneticsPathologyRecombinant DNACRISPR and Genetic EngineeringVirus-based gene therapy researchRetinal Development and Disorders