scholarly journals Recombinant Adeno-associated Virus-mediated Gene Delivery to the Central Nervous System

2007 ◽  
Vol 74 (3) ◽  
pp. 188-189 ◽  
Author(s):  
Toshiyuki Kurai ◽  
Takashi Shimada
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Delivery ◽  
Adeno Associated Virus ◽  
The Central Nervous System

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.

A multicellular, neuro-mimetic model to study nanoparticle uptake in cells of the central nervous system

2014 ◽  
Vol 6 (9) ◽  
pp. 855-861 ◽  
Author(s):  
A. R. Fernandes ◽  
D. M. Chari
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Stem Cell ◽  
Gene Delivery ◽  
Neural Model ◽  
Cell Populations ◽  
Nanoparticle Uptake ◽  
System P ◽  
The Central Nervous System ◽  
In Cells

We describe a multicellular neural model to study nanoparticle uptake and gene delivery, using stem cell derived cell populations.

Nonviral Gene Delivery to the Central Nervous System Based on a Novel Integrin-Targeting Multifunctional Protein

2003 ◽  
Vol 14 (13) ◽  
pp. 1215-1223 ◽  
Author(s):  
H. Peluffo ◽  
A. Arís ◽  
L. Acarin ◽  
B. González ◽  
A. Villaverde ◽  
...  
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Delivery ◽  
Nonviral Gene Delivery ◽  
Multifunctional Protein ◽  
The Central Nervous System

Herpes Simplex Virus as a Gene-Delivery Vector for the Central Nervous System

Viral Vectors ◽  
1995 ◽  
pp. 1-23 ◽  
Author(s):  
Joseph C. Glorioso ◽  
Mary Ann Bender ◽  
William F. Goins ◽  
Neal DeLuca ◽  
David J. Fink
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Herpes Simplex Virus ◽  
Herpes Simplex ◽  
Gene Delivery ◽  
Gene Delivery Vector ◽  
Delivery Vector ◽  
The Central Nervous System ◽  
Simplex Virus

scholarly journals Inhibiting AIDS in the central nervous system: gene delivery to protect neurons from HIV

2003 ◽  
Vol 7 (6) ◽  
pp. 801-810 ◽  
Author(s):  
Pierre Cordelier ◽  
Elisabeth Van Bockstaele ◽  
Sandra A Calarota ◽  
David S Strayer
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Delivery ◽  
The Central Nervous System

scholarly journals Viral vectors for gene delivery to the central nervous system

2012 ◽  
Vol 48 (2) ◽  
pp. 179-188 ◽  
Author(s):  
Thomas B. Lentz ◽  
Steven J. Gray ◽  
R. Jude Samulski
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Delivery ◽  
Viral Vectors ◽  
The Central Nervous System

scholarly journals Non-Viral Delivery of RNA Gene Therapy to the Central Nervous System

Pharmaceutics ◽  
2022 ◽  
Vol 14 (1) ◽  
pp. 165
Author(s):  
Ellen S. Hauck ◽  
James G. Hecker
Keyword(s):  
Gene Therapy ◽  
Central Nervous System ◽  
Nervous System ◽  
Nucleic Acid ◽  
Gene Delivery ◽  
Dna Delivery ◽  
Clinical Applications ◽  
Cationic Lipids ◽  
Viral Dna ◽  
The Central Nervous System

Appropriate gene delivery systems are essential for successful gene therapy in clinical medicine. Lipid-mediated nucleic acid delivery is an alternative to viral vector-mediated gene delivery and has the following advantages. Lipid-mediated delivery of DNA or mRNA is usually more rapid than viral-mediated delivery, offers a larger payload, and has a nearly zero risk of incorporation. Lipid-mediated delivery of DNA or RNA is therefore preferable to viral DNA delivery in those clinical applications that do not require long-term expression for chronic conditions. Delivery of RNA may be preferable to non-viral DNA delivery in some clinical applications, since transit across the nuclear membrane is not necessary, and onset of expression with RNA is therefore even faster than with DNA, although both are faster than most viral vectors. Delivery of RNA to target organ(s) has previously been challenging due to RNA’s rapid degradation in biological systems, but cationic lipids complexed with RNA, as well as lipid nanoparticles (LNPs), have allowed for delivery and expression of the complexed RNA both in vitro and in vivo. This review will focus on the non-viral lipid-mediated delivery of RNAs, including mRNA, siRNA, shRNA, and microRNA, to the central nervous system (CNS), an organ with at least two unique challenges. The CNS contains a large number of slowly dividing or non-dividing cell types and is protected by the blood brain barrier (BBB). In non-dividing cells, RNA-lipid complexes demonstrated increased transfection efficiency relative to DNA transfection. The efficiency, timing of the onset, and duration of expression after transfection may determine which nucleic acid is best for which proposed therapy. Expression can be seen as soon as 1 h after RNA delivery, but duration of expression has been limited to 5–7 h. In contrast, transfection with a DNA lipoplex demonstrates protein expression within 5 h and lasts as long as several weeks after transfection.

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