Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
Jessica L. Centa, Francine M. Jodelka, Anthony J. Hinrich, Tyler B. Johnson, Joseph Ochaba, Michaela Jackson, Dominik M. Duelli, Jill M. Weimer, Frank Rigo, Michelle L. Hastings
Topics & Concepts
Batten diseaseExonExon skippingLysosomal storage diseaseDiseaseAntisense therapyBiologyMedicineOligonucleotideCancer researchGeneticsInternal medicineGeneAlternative splicingLocked nucleic acidLysosomal Storage Disorders ResearchCalcium signaling and nucleotide metabolismNeurogenetic and Muscular Disorders Research