Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors

2021 ◽  
Vol 32 (19-20) ◽  
pp. 1096-1119
Author(s):  
Josephine Macdonald ◽  
Jennifer Marx ◽  
Hildegard Büning
Keyword(s):  
Gene Therapy ◽  
Central Nervous System ◽  
Nervous System ◽  
Adeno Associated Virus ◽  
Virus Vectors

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
Keyword(s):  
Gene Therapy ◽  
Central Nervous System ◽  
Nervous System ◽  
Methylmalonic Acidemia ◽  
Adeno Associated Virus ◽  
Virus Gene

Gene Transfer by Adeno-Associated Virus Vectors into the Central Nervous System

1997 ◽  
Vol 144 (1) ◽  
pp. 113-124 ◽  
Author(s):  
Xiao Xiao ◽  
Juan Li ◽  
Thomas J. McCown ◽  
R.Jude Samulski
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Transfer ◽  
Adeno Associated Virus ◽  
Virus Vectors ◽  
The Central Nervous System

Adeno-Associated Virus Vector for Central Nervous System Gene Therapy

2021 ◽  
Author(s):  
Danqing Zhu ◽  
Adam J. Schieferecke ◽  
Paola A. Lopez ◽  
David V. Schaffer
Keyword(s):  
Gene Therapy ◽  
Central Nervous System ◽  
Nervous System ◽  
Virus Vector ◽  
Adeno Associated Virus

scholarly journals Positron emission tomography reporter gene strategy for use in the central nervous system

2019 ◽  
Vol 116 (23) ◽  
pp. 11402-11407 ◽  
Author(s):  
Tom Haywood ◽  
Corinne Beinat ◽  
Gayatri Gowrishankar ◽  
Chirag B. Patel ◽  
Israt S. Alam ◽  
...  
Keyword(s):  
Positron Emission Tomography ◽  
Central Nervous System ◽  
Nervous System ◽  
Reporter Gene ◽  
Pet Imaging ◽  
Emission Tomography ◽  
Great Promise ◽  
Adeno Associated Virus ◽  
Positron Emission ◽  
The Central Nervous System

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.

Adeno-associated virus vectors for human gene therapy

Gene Therapy ◽  
2020 ◽  
pp. 77-92
Author(s):  
Jeffrey S. Bartlett ◽  
Richard J. Samulski
Keyword(s):  
Gene Therapy ◽  
Human Gene ◽  
Adeno Associated Virus ◽  
Human Gene Therapy ◽  
Virus Vectors
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