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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Autores: 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
Tipo de recurso: artículo
Fecha de publicación:2022
País:España
Institución:Asociación Española de Audiología
Repositorio:Dadun. Depósito Académico Digital de la Universidad de Navarra
Idioma:inglés
OAI Identifier:oai:dadun.unav.edu:10171/118490
Acceso en línea:https://hdl.handle.net/10171/118490
Access Level:acceso abierto
Palabra clave:AAV
Gene therapy
Disease-modifying therapeutics
Neuroprotection
Precision medicine
Descripción
Sumario: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.