Intrathecal delivery of fluorescent labeled butyrylcholinesterase to the brains of butyrylcholinesterase knock-out mice: Visualization and quantification of enzyme distribution in the brain

Global diffuse distribution in the brain

Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1

✍ Naotaka Iwamoto; Atsushi Watanabe; Motoko Yamamoto; Noriko Miyake; Toshiyuki Kur 📂 Article 📅 2009 🏛 John Wiley and Sons 🌐 English ⚖ 383 KB

## Abstract ## Background The success of gene therapy for inherited neurodegenerative diseases such as metachromatic leukodystrophy (MLD) depends on the development of efficient gene delivery throughout the brain guarded by the blood–brain barrier and achieves distribution of the deficient enzyme