Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies
Background: Hearing impairment is the most frequent sensory deficit, affecting 466 million people worldwide and has been listed by the World Health Organization (WHO) as one of the priority diseases for research into therapeutic interventions to address public health needs. Inner ear gene therapy is...
Main Authors: | , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
Frontiers Media S.A.
2021-08-01
|
Series: | Frontiers in Cellular Neuroscience |
Subjects: | |
Online Access: | https://www.frontiersin.org/articles/10.3389/fncel.2021.728610/full |
id |
doaj-908af21b15524884bf9f765dfa0d871b |
---|---|
record_format |
Article |
spelling |
doaj-908af21b15524884bf9f765dfa0d871b2021-09-03T17:44:50ZengFrontiers Media S.A.Frontiers in Cellular Neuroscience1662-51022021-08-011510.3389/fncel.2021.728610728610Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal StudiesDorien Verdoodt0Dorien Verdoodt1Noa Peeleman2Guy Van Camp3Vincent Van Rompaey4Vincent Van Rompaey5Peter Ponsaerts6Department of Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, BelgiumLaboratory of Experimental Hematology, Vaccine and Infectious Disease Institute (Vaxinfectio), University of Antwerp, Antwerp, BelgiumDepartment of Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, BelgiumDepartment of Medical Genetics, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, BelgiumDepartment of Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, BelgiumDepartment of Otorhinolaryngology and Head & Neck Surgery, Antwerp University Hospital, Antwerp, BelgiumLaboratory of Experimental Hematology, Vaccine and Infectious Disease Institute (Vaxinfectio), University of Antwerp, Antwerp, BelgiumBackground: Hearing impairment is the most frequent sensory deficit, affecting 466 million people worldwide and has been listed by the World Health Organization (WHO) as one of the priority diseases for research into therapeutic interventions to address public health needs. Inner ear gene therapy is a promising approach to restore sensorineural hearing loss, for which several gene therapy applications have been studied and reported in preclinical animal studies.Objective: To perform a systematic review on preclinical studies reporting cochlear gene therapy, with a specific focus on transduction efficiency.Methods: An initial PubMed search was performed on April 1st 2021 using the PRISMA methodology. Preclinical in vivo studies reporting primary data regarding transduction efficiency of gene therapy targeting the inner ear were included in this report.Results: Thirty-six studies were included in this review. Transduction of various cell types in the inner ear can be achieved, according to the viral vector used. However, there is significant variability in the applied vector delivery systems, including promoter, viral vector titer, etc.Conclusion: Although gene therapy presents a promising approach to treat sensorineural hearing loss in preclinical studies, the heterogeneity of methodologies impedes the identification of the most promising tools for future use in inner ear therapies.https://www.frontiersin.org/articles/10.3389/fncel.2021.728610/fulltransduction efficiencyinner earadeno-associated virusgene therapyimmunogenicity |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Dorien Verdoodt Dorien Verdoodt Noa Peeleman Guy Van Camp Vincent Van Rompaey Vincent Van Rompaey Peter Ponsaerts |
spellingShingle |
Dorien Verdoodt Dorien Verdoodt Noa Peeleman Guy Van Camp Vincent Van Rompaey Vincent Van Rompaey Peter Ponsaerts Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies Frontiers in Cellular Neuroscience transduction efficiency inner ear adeno-associated virus gene therapy immunogenicity |
author_facet |
Dorien Verdoodt Dorien Verdoodt Noa Peeleman Guy Van Camp Vincent Van Rompaey Vincent Van Rompaey Peter Ponsaerts |
author_sort |
Dorien Verdoodt |
title |
Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies |
title_short |
Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies |
title_full |
Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies |
title_fullStr |
Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies |
title_full_unstemmed |
Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies |
title_sort |
transduction efficiency and immunogenicity of viral vectors for cochlear gene therapy: a systematic review of preclinical animal studies |
publisher |
Frontiers Media S.A. |
series |
Frontiers in Cellular Neuroscience |
issn |
1662-5102 |
publishDate |
2021-08-01 |
description |
Background: Hearing impairment is the most frequent sensory deficit, affecting 466 million people worldwide and has been listed by the World Health Organization (WHO) as one of the priority diseases for research into therapeutic interventions to address public health needs. Inner ear gene therapy is a promising approach to restore sensorineural hearing loss, for which several gene therapy applications have been studied and reported in preclinical animal studies.Objective: To perform a systematic review on preclinical studies reporting cochlear gene therapy, with a specific focus on transduction efficiency.Methods: An initial PubMed search was performed on April 1st 2021 using the PRISMA methodology. Preclinical in vivo studies reporting primary data regarding transduction efficiency of gene therapy targeting the inner ear were included in this report.Results: Thirty-six studies were included in this review. Transduction of various cell types in the inner ear can be achieved, according to the viral vector used. However, there is significant variability in the applied vector delivery systems, including promoter, viral vector titer, etc.Conclusion: Although gene therapy presents a promising approach to treat sensorineural hearing loss in preclinical studies, the heterogeneity of methodologies impedes the identification of the most promising tools for future use in inner ear therapies. |
topic |
transduction efficiency inner ear adeno-associated virus gene therapy immunogenicity |
url |
https://www.frontiersin.org/articles/10.3389/fncel.2021.728610/full |
work_keys_str_mv |
AT dorienverdoodt transductionefficiencyandimmunogenicityofviralvectorsforcochleargenetherapyasystematicreviewofpreclinicalanimalstudies AT dorienverdoodt transductionefficiencyandimmunogenicityofviralvectorsforcochleargenetherapyasystematicreviewofpreclinicalanimalstudies AT noapeeleman transductionefficiencyandimmunogenicityofviralvectorsforcochleargenetherapyasystematicreviewofpreclinicalanimalstudies AT guyvancamp transductionefficiencyandimmunogenicityofviralvectorsforcochleargenetherapyasystematicreviewofpreclinicalanimalstudies AT vincentvanrompaey transductionefficiencyandimmunogenicityofviralvectorsforcochleargenetherapyasystematicreviewofpreclinicalanimalstudies AT vincentvanrompaey transductionefficiencyandimmunogenicityofviralvectorsforcochleargenetherapyasystematicreviewofpreclinicalanimalstudies AT peterponsaerts transductionefficiencyandimmunogenicityofviralvectorsforcochleargenetherapyasystematicreviewofpreclinicalanimalstudies |
_version_ |
1717815976408383488 |