Adeno-Associated Viral Vectors for Clinical Gene Therapy in the Brain

2005 ◽  
pp. 154-168 ◽  
Author(s):  
R. Jude Samulski ◽  
Jennifer Giles
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Clinical Gene Therapy ◽  
The Brain

Gene Therapy, A Novel Therapeutic Tool for Neurological Disorders: Current Progress, Challenges and Future Prospective

2020 ◽  
Vol 20 (3) ◽  
pp. 184-194 ◽  
Author(s):  
Ashif Iqubal ◽  
Mohammad Kashif Iqubal ◽  
Aamir Khan ◽  
Javed Ali ◽  
Sanjula Baboota ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Targeted Delivery ◽  
Neurological Disorders ◽  
Viral Vectors ◽  
Gene Selection ◽  
Current Status ◽  
Lysosomal Storage ◽  
Molecular Etiology ◽  
The Brain ◽  
Lateral Sclerosis

: Neurological disorders are one of the major threat for health care system as they put enormous socioeconomic burden. All aged populations are susceptible to one or other neurological problems with symptoms of neuroinflammation, neurodegeneration and cognitive dysfunction. At present, available pharmacotherapeutics are insufficient to treat these diseased conditions and in most cases, they provide only palliative effect. It was also found that the molecular etiology of neurological disorders is directly linked with the alteration in genetic makeup, which can be inherited or triggered by the injury, environmental toxins and by some existing disease. Therefore, to take care of this situation, gene therapy has emerged as an advanced modality that claims to permanently cure the disease by deletion, silencing or edition of faulty genes and by insertion of healthier genes. In this modality, vectors (viral and non-viral) are used to deliver targeted gene into a specific region of the brain via various routes. At present, gene therapy has shown positive outcomes in complex neurological disorders, such as Parkinson's disease, Alzheimer's disease, Huntington disease, Multiple sclerosis, Amyotrophic lateral sclerosis and in lysosomal storage disease. However, there are some limitations such as immunogenic reactions non-specificity of viral vectors and a lack of effective biomarkers to understand the efficacy of therapy. Considerable progress has been made to improve vector design, gene selection and targeted delivery. This review article deals with the current status of gene therapy in neurological disorders along with its clinical relevance, challenges and future prospective.

scholarly journals Gene therapy using viral vectors for acute neurologic insults

2000 ◽  
Vol XXXII (1-2) ◽  
pp. 82-82
Author(s):  
R. Sapolsky ◽  
G. Steinberg
Keyword(s):  
Gene Therapy ◽  
Brain Tissue ◽  
Viral Vectors ◽  
Molecular Processes ◽  
Hypoxia Ischemia ◽  
The Brain

Currently, many studies are devoted to the cellular and molecular processes occurring during the death of neurons due to hypoxia - ischemia of the brain tissue.

Gene therapy based on lipid nanoparticles as non-viral vectors for Gliomas treatment

2020 ◽  
Vol 20 ◽  
Author(s):  
Marcela Tavares Luiz ◽  
Larissa Bueno Tofani ◽  
Victor Hugo Sousa Araújo ◽  
Leonardo Delello Di Filippo ◽  
Jonatas Lobato Duarte ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Multidrug Resistance ◽  
Viral Vectors ◽  
Lipid Nanoparticles ◽  
Resistance Mechanisms ◽  
Target Cells ◽  
Promising Strategy ◽  
Cellular Apoptosis ◽  
The Brain ◽  
Astrocytoma Grade Iv

Abstract:: Gliomas are primary brain tumors originating from glial cells, representing 30% of all Central Nervous System (CNS) neoplasia. Among them, the astrocytoma grade IV (glioblastoma multiforme) is the most common presenting an invasive and aggressive profile, with an estimated life expectancy about 15 months after diagnosis even after treatment with radiation, surgical resection, and chemotherapy. This poor prognostic is related to the presence of the blood-brain barrier (BBB) and multidrug resistance mechanisms that avoid the uptake and retention of chemotherapeutics inside the brain. Gene therapy has been a promising strategy to overcome these treatment limitations since it has the ability to modify the defective genetic information in tumor cells, being able to induce cellular apoptosis, and silencing genes responsible for multidrug resistance. Lipid-based nanoparticles, non-viral vectors, have been investigated to deliver genes across the BBB to reach the gliomas cells target. Besides that, its low immunogenicity, easy production, ability in the incorporation of ligands to specific target cells and capacity to carry higher size genes, has become the gene therapy based on non-viral vectors promising glioma treatment. In this context, this review will address the most common non-viral vectors based on lipid-based nanoparticles used for gliomas gene therapy such as liposomes, solid lipid nanoparticles, nanostructured lipid carriers and nanoemulsions.

Development of Safe and Efficient Viral Vectors for Clinical Gene Therapy

2009 ◽  
Vol 1 (1) ◽  
pp. 35-41 ◽  
Author(s):  
Jani K. Räty ◽  
Hanna P. Lesch ◽  
Seppo Ylä-Herttuala
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Clinical Gene Therapy

Non-Viral Vectors for Gene Delivery

2018 ◽  
Vol 9 (1) ◽  
pp. 4-11 ◽  
Author(s):  
Aparna Bansal ◽  
Himanshu
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Expression System ◽  
Target Cells ◽  
Specific Expression ◽  
Naked Dna

Introduction: Gene therapy has emerged out as a promising therapeutic pave for the treatment of genetic and acquired diseases. Gene transfection into target cells using naked DNA is a simple and safe approach which has been further improved by combining vectors or gene carriers. Both viral and non-viral approaches have achieved a milestone to establish this technique, but non-viral approaches have attained a significant attention because of their favourable properties like less immunotoxicity and biosafety, easy to produce with versatile surface modifications, etc. Literature is rich in evidences which revealed that undoubtedly, non–viral vectors have acquired a unique place in gene therapy but still there are number of challenges which are to be overcome to increase their effectiveness and prove them ideal gene vectors. Conclusion: To date, tissue specific expression, long lasting gene expression system, enhanced gene transfection efficiency has been achieved with improvement in delivery methods using non-viral vectors. This review mainly summarizes the various physical and chemical methods for gene transfer in vitro and in vivo.

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