Treatment of a Mouse Model of ALS by In Vivo Base Editing
Colin K.W. Lim, Michael Gapinske, Alexandra K. Brooks, Wendy S. Woods, Jackson E. Powell, M. Alejandra Zeballos C., Jackson Winter, Pablo Pérez‐Piñera, Thomas Gaj
Topics & Concepts
BiologyGenetic enhancementMutantSOD1Nonsense mutationGenome editingGene targetingIn vivoGeneCancer researchCRISPRMolecular biologyGeneticsMutationMissense mutationCRISPR and Genetic EngineeringViral Infections and Immunology ResearchAmyotrophic Lateral Sclerosis Research