Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing
Steven Erwood, Onofrio Laselva, Teija M.I. Bily, Reid A. Brewer, Alexandra H. Rutherford, Christine E. Bear, Evgueni A. Ivakine
Abstract
gene or applied as a generalized approach for gene-specific prevention of NMD in disorders where a truncated protein product retains full or partial functionality.
Topics & Concepts
Cystic fibrosisNonsenseAlleleGeneticsGenome editingGenomeHomology (biology)BiologyComputational biologyEvolutionary biologyGeneCystic Fibrosis Research AdvancesCRISPR and Genetic EngineeringAdvanced biosensing and bioanalysis techniques