Gene Therapy in Neuropathy
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Genetics and Genomics".
Deadline for manuscript submissions: 20 November 2024 | Viewed by 659
Special Issue Editor
Special Issue Information
Dear Colleagues,
Treatment of neurogenetic diseases has been supportive and symptomatic with various clinical signs including muscle weakness, slowing of nerve conduction velocities, shaking, developmental delay, and hypotonia. Recent discoveries of molecular mechanisms responsible for the development of these diseases have led to promising new therapies. Gene therapy approaches have been developed in recent decades; nevertheless, there are still some limitations to their extensive use in the clinic. Emerging therapies include the use of viral and nonviral approaches and different routes of administration for gene addition, silencing, replacement, modification, or repair, aiming for phenotypic improvement.
In this Special Issue of the International Journal of Molecular Sciences, we will provide an overview of the different gene therapy strategies developed for central and peripheral nervous system neuropathies. Limitations as well as further steps for clinical applications will be discussed in this Special Issue.
Dr. Alexia Kagiava
Guest Editor
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Keywords
- gene therapy
- adeno-associated viral vectors
- lentiviral vectors
- nanoparticles
- antisense oligonucleotides
- CRISPR Cas9
- RNA
- CNS
- PNS
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