scholarly journals Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia

2021 ◽  
Vol 21 ◽  
pp. 765-776
Author(s):  
Francis J. May ◽  
PamelaSara E. Head ◽  
Leah E. Venturoni ◽  
Randy J. Chandler ◽  
Charles P. Venditti
2021 ◽  
Vol 32 (19-20) ◽  
pp. 1096-1119
Author(s):  
Josephine Macdonald ◽  
Jennifer Marx ◽  
Hildegard Büning

Author(s):  
Danqing Zhu ◽  
Adam J. Schieferecke ◽  
Paola A. Lopez ◽  
David V. Schaffer

Author(s):  
Jared S. Bee ◽  
Kristin O'Berry ◽  
Yu (Zoe) Zhang ◽  
Megan Kuhn Phillippi ◽  
Akanksha Kaushal ◽  
...  

2019 ◽  
Vol 116 (23) ◽  
pp. 11402-11407 ◽  
Author(s):  
Tom Haywood ◽  
Corinne Beinat ◽  
Gayatri Gowrishankar ◽  
Chirag B. Patel ◽  
Israt S. Alam ◽  
...  

There is a growing need for monitoring or imaging gene therapy in the central nervous system (CNS). This can be achieved with a positron emission tomography (PET) reporter gene strategy. Here we report the development of a PET reporter gene system using the PKM2 gene with its associated radiotracer [18F]DASA-23. The PKM2 reporter gene was delivered to the brains of mice by adeno-associated virus (AAV9) via stereotactic injection. Serial PET imaging was carried out over 8 wk to assess PKM2 expression. After 8 wk, the brains were excised for further mRNA and protein analysis. PET imaging at 8 wk post-AAV delivery showed an increase in [18F]DASA-23 brain uptake in the transduced site of mice injected with the AAV mice over all controls. We believe PKM2 shows great promise as a PET reporter gene and to date is the only example that can be used in all areas of the CNS without breaking the blood–brain barrier, to monitor gene and cell therapy.


2015 ◽  
Vol 125 (2) ◽  
pp. 870-880 ◽  
Author(s):  
Randy J. Chandler ◽  
Matthew C. LaFave ◽  
Gaurav K. Varshney ◽  
Niraj S. Trivedi ◽  
Nuria Carrillo-Carrasco ◽  
...  

Neurosurgery ◽  
2001 ◽  
Vol 48 (3) ◽  
pp. 463-475 ◽  
Author(s):  
L. Nelson Hopkins ◽  
Giuseppe Lanzino ◽  
Lee R. Guterman

Abstract IN THE PAST few decades, dramatic improvements have occurred in the field of neuroendovascular surgery. Endovascular therapy today is a well-established treatment modality for a variety of cerebrovascular and nonvascular central nervous system diseases. The foundation of this spectacular evolution was laid by the efforts of pioneering visionaries who often worked alone and under difficult, almost impossible, conditions. Ongoing device development and refinement have revolutionized the field at a dizzying, exhilarating pace. With a better understanding of the molecular basis of diseases and further advancements in gene therapy, neuroendovascular techniques have an enormous potential for application to the entire spectrum of central nervous system diseases as a minimally invasive vehicle for the delivery of biological factors.


2004 ◽  
Vol 9 (1) ◽  
pp. 56-66 ◽  
Author(s):  
Alshad S Lalani ◽  
Betty Chang ◽  
JianMin Lin ◽  
Scott S Case ◽  
Bo Luan ◽  
...  

Sign in / Sign up

Export Citation Format

Share Document