Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing <i>CLN2</i>
Dolan Sondhi, Stephen M. Kaminsky, Neil R. Hackett, Odelya E. Pagovich, Jonathan B. Rosenberg, Bishnu P. De, Alvin Chen, Benjamin Van de Graaf, Jason G. Mezey, Grace W. Mammen, Denesy Mancenido, Fang Xu, Barry E. Kosofsky, Kaleb Yohay, Stefan Worgall, Robert J. Kaner, Mark Souwedaine, Bruce M. Greenwald, Michael G. Kaplitt, Jonathan P. Dyke, Douglas Ballon, Linda Heier, Szilárd Kiss, Ronald G. Crystal
Abstract
< 0.0001), respectively. Intraparenchymal brain administration of AAVrh.10hCLN2 slowed the progression of disease in children with CLN2 disease. However, improvements in vector design and delivery strategies will be necessary to halt disease progression using gene therapy.
Topics & Concepts
ParenchymaVirusAdeno-associated virusMedicineDiseaseVirologyPathologyGeneBiologyGeneticsRecombinant DNAVector (molecular biology)Lysosomal Storage Disorders ResearchCytomegalovirus and herpesvirus researchRNA regulation and disease