Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives

It is without doubt that the gene therapy field is currently in the spotlight for the development of new therapeutics targeting unmet medical needs. Thus, considering the gene therapy scenario, neurological diseases in general and neurodegenerative disorders in particular are emerging as the most ap...

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Authors: Fajardo-Serrano, A. (Ana)|||/items/d93bf181-23cd-4267-98ec-609cd7a5a9fe, Rico, A.J. (Alberto J.)|||/items/7a177958-067e-4900-943e-0271d63a5021, Roda, E. (Elvira)|||/items/7ea91585-d2d9-4d11-aabf-4daab728ca1c, Honrubia, A. (Adriana)|||/items/99298a39-e5c4-4985-a5db-f0eaa5863aaf, Arrieta, S. (Sandra)|||/items/d8b5e9fa-4b6e-4bd5-a760-deaa9f000f3a, Ariznabarreta, G. (Goiaz)|||/items/1503f072-d975-41fd-8b31-76bbaf4d3da1, Chocarro, J. (Julia)|||/items/1c162220-814c-4baf-9e72-4fa870e90680, Lorenzo-Ramos, E. (Elena)|||/items/6c5a2c66-21e4-43c2-8dc8-f5bda58cb539, Pejenaute-Martínez-de-Lizarrondo, Á. (Álvaro)|||/items/f10e17ed-8b4e-4d5c-8985-3f73a691f9d0, Vazquez, A. (Alfonso)|||/items/7d5cb87e-5c97-4865-aea8-a4694ef0139f, Lanciego-Pérez, J.L. (José Luis)|||/items/d2d4dbfe-4daf-4103-89ec-2ebf41b65cd1
Format: article
Publication Date:2022
Country:España
Institution:Asociación Española de Audiología
Repository:Dadun. Depósito Académico Digital de la Universidad de Navarra
Language:English
OAI Identifier:oai:dadun.unav.edu:10171/118490
Online Access:https://hdl.handle.net/10171/118490
Access Level:Open access
Keyword:AAV
Gene therapy
Disease-modifying therapeutics
Neuroprotection
Precision medicine
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spelling Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectivesFajardo-Serrano, A. (Ana)|||/items/d93bf181-23cd-4267-98ec-609cd7a5a9feRico, A.J. (Alberto J.)|||/items/7a177958-067e-4900-943e-0271d63a5021Roda, E. (Elvira)|||/items/7ea91585-d2d9-4d11-aabf-4daab728ca1cHonrubia, A. (Adriana)|||/items/99298a39-e5c4-4985-a5db-f0eaa5863aafArrieta, S. (Sandra)|||/items/d8b5e9fa-4b6e-4bd5-a760-deaa9f000f3aAriznabarreta, G. (Goiaz)|||/items/1503f072-d975-41fd-8b31-76bbaf4d3da1Chocarro, J. (Julia)|||/items/1c162220-814c-4baf-9e72-4fa870e90680Lorenzo-Ramos, E. (Elena)|||/items/6c5a2c66-21e4-43c2-8dc8-f5bda58cb539Pejenaute-Martínez-de-Lizarrondo, Á. (Álvaro)|||/items/f10e17ed-8b4e-4d5c-8985-3f73a691f9d0Vazquez, A. (Alfonso)|||/items/7d5cb87e-5c97-4865-aea8-a4694ef0139fLanciego-Pérez, J.L. (José Luis)|||/items/d2d4dbfe-4daf-4103-89ec-2ebf41b65cd1AAVGene therapyDisease-modifying therapeuticsNeuroprotectionPrecision medicineIt is without doubt that the gene therapy field is currently in the spotlight for the development of new therapeutics targeting unmet medical needs. Thus, considering the gene therapy scenario, neurological diseases in general and neurodegenerative disorders in particular are emerging as the most appealing choices for new therapeutic arrivals intended to slow down, stop, or even revert the natural progressive course that characterizes most of these devastating neurodegenerative processes. Since an extensive coverage of all available literature is not feasible in practical terms, here emphasis was made in providing some advice to beginners in the field with a narrow focus on elucidating the best delivery route available for fulfilling any given AAV-based therapeutic approach. Furthermore, it is worth nothing that the number of ongoing clinical trials is increasing at a breathtaking speed. Accordingly, a landscape view of preclinical and clinical initiatives is also provided here in an attempt to best illustrate what is ongoing in this quickly expanding field.MDPIDadun. Depósito Académico Digital Universidad de Navarra20222022-01-0120222022-01-01journal articlehttp://purl.org/coar/resource_type/c_6501info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/10171/118490reponame:Dadun. Depósito Académico Digital de la Universidad de Navarrainstname:Asociación Española de AudiologíaInglésengopen accesshttp://purl.org/coar/access_right/c_abf2info:eu-repo/semantics/openAccessoai:dadun.unav.edu:10171/1184902026-06-21T12:47:57Z
dc.title.none.fl_str_mv Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
title Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
spellingShingle Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
Fajardo-Serrano, A. (Ana)|||/items/d93bf181-23cd-4267-98ec-609cd7a5a9fe
AAV
Gene therapy
Disease-modifying therapeutics
Neuroprotection
Precision medicine
title_short Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
title_full Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
title_fullStr Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
title_full_unstemmed Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
title_sort Adeno-associated viral vectors as versatile tools for neurological disorders: focus on delivery routes and therapeutic perspectives
dc.creator.none.fl_str_mv Fajardo-Serrano, A. (Ana)|||/items/d93bf181-23cd-4267-98ec-609cd7a5a9fe
Rico, A.J. (Alberto J.)|||/items/7a177958-067e-4900-943e-0271d63a5021
Roda, E. (Elvira)|||/items/7ea91585-d2d9-4d11-aabf-4daab728ca1c
Honrubia, A. (Adriana)|||/items/99298a39-e5c4-4985-a5db-f0eaa5863aaf
Arrieta, S. (Sandra)|||/items/d8b5e9fa-4b6e-4bd5-a760-deaa9f000f3a
Ariznabarreta, G. (Goiaz)|||/items/1503f072-d975-41fd-8b31-76bbaf4d3da1
Chocarro, J. (Julia)|||/items/1c162220-814c-4baf-9e72-4fa870e90680
Lorenzo-Ramos, E. (Elena)|||/items/6c5a2c66-21e4-43c2-8dc8-f5bda58cb539
Pejenaute-Martínez-de-Lizarrondo, Á. (Álvaro)|||/items/f10e17ed-8b4e-4d5c-8985-3f73a691f9d0
Vazquez, A. (Alfonso)|||/items/7d5cb87e-5c97-4865-aea8-a4694ef0139f
Lanciego-Pérez, J.L. (José Luis)|||/items/d2d4dbfe-4daf-4103-89ec-2ebf41b65cd1
author Fajardo-Serrano, A. (Ana)|||/items/d93bf181-23cd-4267-98ec-609cd7a5a9fe
author_facet Fajardo-Serrano, A. (Ana)|||/items/d93bf181-23cd-4267-98ec-609cd7a5a9fe
Rico, A.J. (Alberto J.)|||/items/7a177958-067e-4900-943e-0271d63a5021
Roda, E. (Elvira)|||/items/7ea91585-d2d9-4d11-aabf-4daab728ca1c
Honrubia, A. (Adriana)|||/items/99298a39-e5c4-4985-a5db-f0eaa5863aaf
Arrieta, S. (Sandra)|||/items/d8b5e9fa-4b6e-4bd5-a760-deaa9f000f3a
Ariznabarreta, G. (Goiaz)|||/items/1503f072-d975-41fd-8b31-76bbaf4d3da1
Chocarro, J. (Julia)|||/items/1c162220-814c-4baf-9e72-4fa870e90680
Lorenzo-Ramos, E. (Elena)|||/items/6c5a2c66-21e4-43c2-8dc8-f5bda58cb539
Pejenaute-Martínez-de-Lizarrondo, Á. (Álvaro)|||/items/f10e17ed-8b4e-4d5c-8985-3f73a691f9d0
Vazquez, A. (Alfonso)|||/items/7d5cb87e-5c97-4865-aea8-a4694ef0139f
Lanciego-Pérez, J.L. (José Luis)|||/items/d2d4dbfe-4daf-4103-89ec-2ebf41b65cd1
author_role author
author2 Rico, A.J. (Alberto J.)|||/items/7a177958-067e-4900-943e-0271d63a5021
Roda, E. (Elvira)|||/items/7ea91585-d2d9-4d11-aabf-4daab728ca1c
Honrubia, A. (Adriana)|||/items/99298a39-e5c4-4985-a5db-f0eaa5863aaf
Arrieta, S. (Sandra)|||/items/d8b5e9fa-4b6e-4bd5-a760-deaa9f000f3a
Ariznabarreta, G. (Goiaz)|||/items/1503f072-d975-41fd-8b31-76bbaf4d3da1
Chocarro, J. (Julia)|||/items/1c162220-814c-4baf-9e72-4fa870e90680
Lorenzo-Ramos, E. (Elena)|||/items/6c5a2c66-21e4-43c2-8dc8-f5bda58cb539
Pejenaute-Martínez-de-Lizarrondo, Á. (Álvaro)|||/items/f10e17ed-8b4e-4d5c-8985-3f73a691f9d0
Vazquez, A. (Alfonso)|||/items/7d5cb87e-5c97-4865-aea8-a4694ef0139f
Lanciego-Pérez, J.L. (José Luis)|||/items/d2d4dbfe-4daf-4103-89ec-2ebf41b65cd1
author2_role author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Dadun. Depósito Académico Digital Universidad de Navarra
dc.subject.none.fl_str_mv AAV
Gene therapy
Disease-modifying therapeutics
Neuroprotection
Precision medicine
topic AAV
Gene therapy
Disease-modifying therapeutics
Neuroprotection
Precision medicine
description It is without doubt that the gene therapy field is currently in the spotlight for the development of new therapeutics targeting unmet medical needs. Thus, considering the gene therapy scenario, neurological diseases in general and neurodegenerative disorders in particular are emerging as the most appealing choices for new therapeutic arrivals intended to slow down, stop, or even revert the natural progressive course that characterizes most of these devastating neurodegenerative processes. Since an extensive coverage of all available literature is not feasible in practical terms, here emphasis was made in providing some advice to beginners in the field with a narrow focus on elucidating the best delivery route available for fulfilling any given AAV-based therapeutic approach. Furthermore, it is worth nothing that the number of ongoing clinical trials is increasing at a breathtaking speed. Accordingly, a landscape view of preclinical and clinical initiatives is also provided here in an attempt to best illustrate what is ongoing in this quickly expanding field.
publishDate 2022
dc.date.none.fl_str_mv 2022
2022-01-01
2022
2022-01-01
dc.type.none.fl_str_mv journal article
http://purl.org/coar/resource_type/c_6501
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/10171/118490
url https://hdl.handle.net/10171/118490
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv MDPI
publisher.none.fl_str_mv MDPI
dc.source.none.fl_str_mv reponame:Dadun. Depósito Académico Digital de la Universidad de Navarra
instname:Asociación Española de Audiología
instname_str Asociación Española de Audiología
reponame_str Dadun. Depósito Académico Digital de la Universidad de Navarra
collection Dadun. Depósito Académico Digital de la Universidad de Navarra
repository.name.fl_str_mv
repository.mail.fl_str_mv
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