Adeno-associated virus vector-based gene therapies for pediatric diseases

Gene therapy using adeno-associated virus (AAV) is a rapidly developing technology with widespread treatment potential. AAV2 vectors injected directly into the brain by stereotaxic brain surgery have shown good results in treating aromatic l-amino acid decarboxylase deficiency. Moreover, gene therap...

Full description

Saved in:
Bibliographic Details
Main Authors: Kazuhiro Muramatsu, Shin-ichi Muramatsu
Format: Article
Language:English
Published: Elsevier 2023-02-01
Series:Pediatrics and Neonatology
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S1875957222002133
Tags: Add Tag
No Tags, Be the first to tag this record!