Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space
Year: 2018
Type: article
Abstract: Recombinant adeno-associated viruses (rAAVs) are the leading in vivo gene delivery platform, and have been extensively studied in gene therapy targeting various tissues, including the central nervous system (CNS). A single-bolus rAAV injection to the cerebrospinal fluid (CSF) space has been widely used to target the CNS, but it suffers from several drawbacks, such as leakage to peripheral tissues. Here, a protocol is described using an osmotic pump to infuse rAAV slowly into the mouse CSF space. Compared to the single-bolus injection technique, pump infusion can lead to higher CNS transduction and lower transduction in the peripheral tissues.
Source: Human Gene Therapy Methods
Authors: Dan Wang, Jia Li, Karen Tran, Daniel R. Burt, Li Zhong, Guangping Gao
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
Awards: New Approaches to Gene Therapy for Alpha-1 Antitrypsin Deficiency, Next Generation of Recombinant AAV Serotype For Gene Therapy, A Gene therapeutic approach to stable suppression of HIV-1 replication, Oligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and Leukodystrophies
OpenAlex ID: W2792939569