Abstract
Introduction: The recent approval of Spinraza (nusinersen), an antisense oligonucleotide, by U.S. Food and Drug Administration to treat patients with spinal muscular atrophy, has reignited interests of researchers in designing and testing new gene therapy approaches to treat neurological disorders, in particular, to curb neurodegenerative diseases of the central nervous system which represent an ever-increasing public health burden to today's society.
Conclusion: This review highlights several key factors to be taken into consideration to design successful preclinical and clinical gene therapy experiments with respect to the vehicle of delivery and the route of administration to CNS-specific targets, with an additional focus on antisense oligonucleotide therapy and recent clinical trial developments.
Keywords: Gene therapy, Neurodegenerative disorder, Antisense oligonucleotide, Clinical trial, Viral vectors, Central nervous system.
Current Gene Therapy
Title:Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders
Volume: 17 Issue: 3
Author(s): Aneesh Donde, Philip C. Wong and Liam L. Chen*
Affiliation:
- Department of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21205,United States
Keywords: Gene therapy, Neurodegenerative disorder, Antisense oligonucleotide, Clinical trial, Viral vectors, Central nervous system.
Abstract: Introduction: The recent approval of Spinraza (nusinersen), an antisense oligonucleotide, by U.S. Food and Drug Administration to treat patients with spinal muscular atrophy, has reignited interests of researchers in designing and testing new gene therapy approaches to treat neurological disorders, in particular, to curb neurodegenerative diseases of the central nervous system which represent an ever-increasing public health burden to today's society.
Conclusion: This review highlights several key factors to be taken into consideration to design successful preclinical and clinical gene therapy experiments with respect to the vehicle of delivery and the route of administration to CNS-specific targets, with an additional focus on antisense oligonucleotide therapy and recent clinical trial developments.
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Donde Aneesh , Wong C. Philip and Chen L. Liam *, Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders, Current Gene Therapy 2017; 17 (3) . https://dx.doi.org/10.2174/1566523217666171013124150
DOI https://dx.doi.org/10.2174/1566523217666171013124150 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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