Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy
Nicola Specchio, Alessandro Ferretti, Marina Trivisano, Nicola Pietrafusa, Chiara Pepi, Costanza Calabrese, Susanna Livadiotti, Alessandra Simonetti, Paolo Rossi, Paolo Curatolo, Federico Vigevano
Topics & Concepts
Batten diseaseNeuronal ceroid lipofuscinosisMedicineEnzyme replacement therapyDiseaseDementiaLysosomal storage diseaseAdverse effectGenetic enhancementBioinformaticsNeurosciencePathologyPharmacologyBiologyGeneticsGeneLysosomal Storage Disorders ResearchCellular transport and secretionRetinal Development and Disorders