Interim results from the first-in-human intracisternal dosing of RGX-181 investigational AAV9 gene therapy in a child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)

Interim results from the first-in-human intracisternal dosing of RGX-181 investigational AAV9 gene therapy in a child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)

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Bibliographic Details
Published in:Molecular genetics and metabolism Vol. 141; no. 2; p. 107970
Main Authors: de Souza, Carolina Fischinger Moura, Alves, Tamires, Duarte, Juliana, Burke, Jenna, Chan, Gary, Higgins, Mikayla, Falabella, Paulo, Phillips, Dawn, Ohnsman, Christina, Giugliani, Roberto
Format: Journal Article
Language:English
Published: Elsevier Inc 01-02-2024
Online Access:Get full text
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2023.107970