Future Prospects of Gene Therapy for Treating CNS Diseases

2000 ◽  
pp. 485-508
Author(s):  
Daniel A. Peterson ◽  
Jasodhara Ray ◽  
Fred H. Gage
Keyword(s):  
Gene Therapy ◽  
Future Prospects ◽  
Cns Diseases

scholarly journals Latest Advances of Virology Research Using CRISPR/Cas9-Based Gene-Editing Technology and Its Application to Vaccine Development

Viruses ◽  
2021 ◽  
Vol 13 (5) ◽  
pp. 779
Author(s):  
Man Teng ◽  
Yongxiu Yao ◽  
Venugopal Nair ◽  
Jun Luo
Keyword(s):  
Biological Sciences ◽  
Gene Therapy ◽  
Genetic Engineering ◽  
Gene Function ◽  
Viral Genome ◽  
Gene Editing ◽  
Vaccine Development ◽  
Future Prospects ◽  
Dna Viruses ◽  
Viral Genomes

In recent years, the CRISPR/Cas9-based gene-editing techniques have been well developed and applied widely in several aspects of research in the biological sciences, in many species, including humans, animals, plants, and even in viruses. Modification of the viral genome is crucial for revealing gene function, virus pathogenesis, gene therapy, genetic engineering, and vaccine development. Herein, we have provided a brief review of the different technologies for the modification of the viral genomes. Particularly, we have focused on the recently developed CRISPR/Cas9-based gene-editing system, detailing its origin, functional principles, and touching on its latest achievements in virology research and applications in vaccine development, especially in large DNA viruses of humans and animals. Future prospects of CRISPR/Cas9-based gene-editing technology in virology research, including the potential shortcomings, are also discussed.

scholarly journals Strategy of liver-directed gene therapy: present status and future prospects

1999 ◽  
Vol 19 (4) ◽  
pp. 265-274 ◽  
Author(s):  
Yasushi Shiratori ◽  
Fumihiko Kanai ◽  
Makoto Ohashi ◽  
Masao Omata
Keyword(s):  
Gene Therapy ◽  
Present Status ◽  
Future Prospects

AAV9 is considered the most efficient AAV serotype targeting blood-brain barriers. To enhance effective gene therapy for CNS illnesses, testing novel vectors with more efficient crossing capabilities is vital

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Intravenous Injection ◽  
Peripheral Circulation ◽  
Full Potential ◽  
Cns Diseases ◽  
Aav Vector ◽  
Early Clinical Trial ◽  
Blood Brain ◽  
Efficient Gene ◽  
Trial Outcomes

Although gene therapy for CNS diseases shows promise in cell and animal investigations, most human trials have failed to satisfy the requisite requirements. Finding novel techniques to boost the efficacy of gene therapy in treating CNS diseases is still crucial. A growing number of clinical trials have proved the efficacy and safety of using AAV vectors, making AAV vector research a gene therapy hotspot. However, due to the presence of the BBB, many siRNA and DNA with potential therapeutic value are difficult to transport from peripheral circulation to the brain using AAV vectors, limiting the clinical impact of gene therapy drugs in the CNS and posing a major challenge to the field of CNS gene therapy. In early studies, AAV9 was considered the most effective AAV serotype for getting through the blood-brain barrier and transduction to central nervous system cells following intravenous injection. Aavrh10 isolated from rhesus monkeys was equal to, if not superior to, AAV9. AAV-PHP.B, a newly built capsid, exhibits 40-fold greater efficacy than AAV9 in astrocyte and neuron transduction. AAV-PHP.eB, a modified AAV-PHP.B variety, was identified to retain PHP.B's AAV-capacity to transduce astrocytes while enhancing neuronal transduction. While the four serotypes AAV9, AAVrh10, AAV-PHP.B, and AAV-PHP.eB have been validated to penetrate mice's BBB following intravenous injection, the number of AAV vectors that can do so is low. Moreover, the manner in which AAV vectors penetrate the BBB remains unclear. To promote efficient gene therapy for CNS diseases, it is still important to test new vectors with more efficient crossing abilities and understand their crossing processes. In addition to technical challenges, AAV vectors in treating CNS diseases may be limited by cautious attitudes to innovative treatments. Continued advances in AAV vector research, together with early clinical trial outcomes, might help researchers achieve the full potential of AAV-based CNS disease therapies.

Current Research and Future Prospects for Gene Therapy in Andrology

2006 ◽  
pp. 592-598 ◽  
Author(s):  
Y. Kojima ◽  
S. Sasaki ◽  
K. Kohri
Keyword(s):  
Gene Therapy ◽  
Future Prospects

scholarly journals Gene therapy for CNS diseases – Krabbe disease

Bioimpacts ◽  
2016 ◽  
Vol 6 (2) ◽  
pp. 69-70 ◽  
Author(s):  
Mohammad A. Rafi
Keyword(s):  
Gene Therapy ◽  
Krabbe Disease ◽  
Cns Diseases

scholarly journals Review article: gene therapy, recent developments and future prospects in gastrointestinal oncology

2010 ◽  
Vol 32 (8) ◽  
pp. 953-968 ◽  
Author(s):  
Y. Touchefeu ◽  
K. J. Harrington ◽  
J. P. Galmiche ◽  
G. Vassaux
Keyword(s):  
Gene Therapy ◽  
Review Article ◽  
Future Prospects ◽  
Recent Developments

Gene therapy for muscular dystrophy: current progress and future prospects

2009 ◽  
Vol 9 (7) ◽  
pp. 849-866 ◽  
Author(s):  
Capucine Trollet ◽  
Takis Athanasopoulos ◽  
Linda Popplewell ◽  
Alberto Malerba ◽  
George Dickson
Keyword(s):  
Gene Therapy ◽  
Muscular Dystrophy ◽  
Future Prospects
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