Early transgene expression of GALC enzyme in a phase I/II safety, tolerability and efficacy study of PBKR03 in infants with early infantile Krabbe disease (EIKD) (GALax-C)

Saved in:
Bibliographic Details
Published in:Molecular genetics and metabolism 2023-02, Vol.138 (2), p.106999, Article 106999
Main Authors: Al-Zaidy, Samiah A., Wolf, Nicole I., Jones, Simon, Mallack, Eric J., Waldman, Amy, Rubin, Jennifer P., Bonkowsky, Joshua L., Aran, Adi, Ni, Yan G., Nagilla, Pruthvi, Donahue, Dan, Giugliani, Roberto, Forman, Mark S., Bernard, Geneviève
Format: Article
Language:English
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:
ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2022.106999