Gene Transfer to the Central Nervous System: Current State of the Art of the Viral Vectors

2005 ◽  
Vol 6 (1) ◽  
pp. 13-37 ◽  
Author(s):  
E. Kremer
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Transfer ◽  
Viral Vectors ◽  
State Of The Art ◽  
Current State ◽  
The Central Nervous System

Endovascular Restorative Neurosurgery: A Novel Concept for Molecular and Cellular Therapy of the Nervous System

Neurosurgery ◽  
2003 ◽  
Vol 52 (2) ◽  
pp. 402-413 ◽  
Author(s):  
Arun Paul Amar ◽  
Berislav V. Zlokovic ◽  
Michael L.J. Apuzzo
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Transfer ◽  
Viral Vectors ◽  
Developmental Disorders ◽  
Cellular Therapy ◽  
Genetically Engineered ◽  
Host Cells ◽  
The Central Nervous System ◽  
The Brain

Abstract THE AMALGAM OF molecular biology and neurosurgery offers immense promise for neurorestoration and the management of neurodegenerative deficiencies, developmental disorders, neoplasms, stroke, and trauma. This article summarizes present strategies for and impediments to gene therapy and stem cell therapy of the central nervous system and advances the concept of a potential new approach, namely endovascular restorative neurosurgery. The objectives of gene transfer to the central nervous system are efficient transfection of host cells, selective sustained expression of the transgene, and lack of toxicity or immune excitation. The requisite elements of this process are the identification of candidate diseases, the construction of vehicles for gene transfer, regulated expression, and physical delivery. In the selection of target disorders, the underlying genetic events to be overcome, as well as their spatial and temporal distributions, must be considered. These factors determine the requirements for the physical dispersal of the transgene, the duration of transgene expression, and the quantity of transgene product needed to abrogate the disease phenotype. Vehicles for conveying the transgene to the central nervous system include viral vectors (retroviruses, lentiviruses, adenoviruses, adeno-associated viruses, and herpes simplex virus), liposomes, and genetically engineered cells, including neural stem cells. Delivery of the transgene into the brain presents several challenges, including limited and potentially risky access through the cranium, sensitivity to volumetric changes, restricted diffusion, and the blood-brain barrier. Genetic or cellular therapeutic agents may be injected directly into the brain parenchyma (via stereotaxy or craniotomy), into the cerebrospinal fluid (in the ventricles or cisterns), or into the bloodstream (intravenously or intra-arterially). The advantages of the endovascular route include the potential for widespread distribution, the ability to deliver large volumes, limited perturbation of neural tissue, and the feasibility of repeated administration.

scholarly journals Methods for Gene Transfer to the Central Nervous System

2014 ◽  
pp. 125-197 ◽  
Author(s):  
Boris Kantor ◽  
Rachel M. Bailey ◽  
Keon Wimberly ◽  
Sahana N. Kalburgi ◽  
Steven J. Gray
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Transfer ◽  
The Central Nervous System

scholarly journals Recent Developments in Microfluidic Technologies for Central Nervous System Targeted Studies

Pharmaceutics ◽  
2020 ◽  
Vol 12 (6) ◽  
pp. 542
Author(s):  
Maria Inês Teixeira ◽  
Maria Helena Amaral ◽  
Paulo C. Costa ◽  
Carla M. Lopes ◽  
Dimitrios A. Lamprou
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
State Of The Art ◽  
Physicochemical Characteristics ◽  
Future Perspectives ◽  
Invaluable Tool ◽  
Recent Developments ◽  
The Central Nervous System ◽  
Cns Delivery ◽  
Reproducible Manner

Neurodegenerative diseases (NDs) bear a lot of weight in public health. By studying the properties of the blood-brain barrier (BBB) and its fundamental interactions with the central nervous system (CNS), it is possible to improve the understanding of the pathological mechanisms behind these disorders and create new and better strategies to improve bioavailability and therapeutic efficiency, such as nanocarriers. Microfluidics is an intersectional field with many applications. Microfluidic systems can be an invaluable tool to accurately simulate the BBB microenvironment, as well as develop, in a reproducible manner, drug delivery systems with well-defined physicochemical characteristics. This review provides an overview of the most recent advances on microfluidic devices for CNS-targeted studies. Firstly, the importance of the BBB will be addressed, and different experimental BBB models will be briefly discussed. Subsequently, microfluidic-integrated BBB models (BBB/brain-on-a-chip) are introduced and the state of the art reviewed, with special emphasis on their use to study NDs. Additionally, the microfluidic preparation of nanocarriers and other compounds for CNS delivery has been covered. The last section focuses on current challenges and future perspectives of microfluidic experimentation.

Fetal MRI of the central nervous system: State-of-the-art

2017 ◽  
Vol 93 ◽  
pp. 273-283 ◽  
Author(s):  
Lucia Manganaro ◽  
Silvia Bernardo ◽  
Amanda Antonelli ◽  
Valeria Vinci ◽  
Matteo Saldari ◽  
...  
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
State Of The Art ◽  
Fetal Mri ◽  
System State ◽  
The Central Nervous System

Knock-out mouse for Canavan disease: a model for gene transfer to the central nervous system

2000 ◽  
Vol 2 (3) ◽  
pp. 165-175 ◽  
Author(s):  
Reuben Matalon ◽  
Peter L. Rady ◽  
Kenneth A. Platt ◽  
Henry B. Skinner ◽  
Michael J. Quast ◽  
...  
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Transfer ◽  
Canavan Disease ◽  
Knock Out ◽  
The Central Nervous System ◽  
Knock Out Mouse

scholarly journals Adeno-Associated Viral Vectors Serotype 8 for Cell-Specific Delivery of Therapeutic Genes in the Central Nervous System

2017 ◽  
Vol 11 ◽  
Author(s):  
Diego Pignataro ◽  
Diego Sucunza ◽  
Lucia Vanrell ◽  
Esperanza Lopez-Franco ◽  
Iria G. Dopeso-Reyes ◽  
...  
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Viral Vectors ◽  
The Central Nervous System ◽  
Therapeutic Genes
Sign in / Sign up

Export Citation Format

Share Document