Cargando…

Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders

The majority of monogenic liver diseases are autosomal recessive disorders, with few being sex-related or co-dominant. Although orthotopic liver transplantation (LT) is currently the sole therapeutic option for end-stage patients, such an invasive surgical approach is severely restricted by the lack...

Descripción completa

Detalles Bibliográficos
Autores principales: Ghasemzad, Mahsa, Hashemi, Mahdieh, Lavasani, Zohre Miri, Hossein-khannazer, Nikoo, Bakhshandeh, Haleh, Gramignoli, Roberto, Keshavarz Alikhani, Hani, Najimi, Mustapha, Nikeghbalian, Saman, Vosough, Massoud
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9404740/
https://www.ncbi.nlm.nih.gov/pubmed/36004917
http://dx.doi.org/10.3390/bioengineering9080392
_version_ 1784773706556375040
author Ghasemzad, Mahsa
Hashemi, Mahdieh
Lavasani, Zohre Miri
Hossein-khannazer, Nikoo
Bakhshandeh, Haleh
Gramignoli, Roberto
Keshavarz Alikhani, Hani
Najimi, Mustapha
Nikeghbalian, Saman
Vosough, Massoud
author_facet Ghasemzad, Mahsa
Hashemi, Mahdieh
Lavasani, Zohre Miri
Hossein-khannazer, Nikoo
Bakhshandeh, Haleh
Gramignoli, Roberto
Keshavarz Alikhani, Hani
Najimi, Mustapha
Nikeghbalian, Saman
Vosough, Massoud
author_sort Ghasemzad, Mahsa
collection PubMed
description The majority of monogenic liver diseases are autosomal recessive disorders, with few being sex-related or co-dominant. Although orthotopic liver transplantation (LT) is currently the sole therapeutic option for end-stage patients, such an invasive surgical approach is severely restricted by the lack of donors and post-transplant complications, mainly associated with life-long immunosuppressive regimens. Therefore, the last decade has witnessed efforts for innovative cellular or gene-based therapeutic strategies. Gene therapy is a promising approach for treatment of many hereditary disorders, such as monogenic inborn errors. The liver is an organ characterized by unique features, making it an attractive target for in vivo and ex vivo gene transfer. The current genetic approaches for hereditary liver diseases are mediated by viral or non-viral vectors, with promising results generated by gene-editing tools, such as CRISPR-Cas9 technology. Despite massive progress in experimental gene-correction technologies, limitations in validated approaches for monogenic liver disorders have encouraged researchers to refine promising gene therapy protocols. Herein, we highlighted the most common monogenetic liver disorders, followed by proposed genetic engineering approaches, offered as promising therapeutic modalities.
format Online
Article
Text
id pubmed-9404740
institution National Center for Biotechnology Information
language English
publishDate 2022
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-94047402022-08-26 Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders Ghasemzad, Mahsa Hashemi, Mahdieh Lavasani, Zohre Miri Hossein-khannazer, Nikoo Bakhshandeh, Haleh Gramignoli, Roberto Keshavarz Alikhani, Hani Najimi, Mustapha Nikeghbalian, Saman Vosough, Massoud Bioengineering (Basel) Review The majority of monogenic liver diseases are autosomal recessive disorders, with few being sex-related or co-dominant. Although orthotopic liver transplantation (LT) is currently the sole therapeutic option for end-stage patients, such an invasive surgical approach is severely restricted by the lack of donors and post-transplant complications, mainly associated with life-long immunosuppressive regimens. Therefore, the last decade has witnessed efforts for innovative cellular or gene-based therapeutic strategies. Gene therapy is a promising approach for treatment of many hereditary disorders, such as monogenic inborn errors. The liver is an organ characterized by unique features, making it an attractive target for in vivo and ex vivo gene transfer. The current genetic approaches for hereditary liver diseases are mediated by viral or non-viral vectors, with promising results generated by gene-editing tools, such as CRISPR-Cas9 technology. Despite massive progress in experimental gene-correction technologies, limitations in validated approaches for monogenic liver disorders have encouraged researchers to refine promising gene therapy protocols. Herein, we highlighted the most common monogenetic liver disorders, followed by proposed genetic engineering approaches, offered as promising therapeutic modalities. MDPI 2022-08-15 /pmc/articles/PMC9404740/ /pubmed/36004917 http://dx.doi.org/10.3390/bioengineering9080392 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Ghasemzad, Mahsa
Hashemi, Mahdieh
Lavasani, Zohre Miri
Hossein-khannazer, Nikoo
Bakhshandeh, Haleh
Gramignoli, Roberto
Keshavarz Alikhani, Hani
Najimi, Mustapha
Nikeghbalian, Saman
Vosough, Massoud
Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders
title Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders
title_full Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders
title_fullStr Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders
title_full_unstemmed Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders
title_short Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders
title_sort novel gene-correction-based therapeutic modalities for monogenic liver disorders
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9404740/
https://www.ncbi.nlm.nih.gov/pubmed/36004917
http://dx.doi.org/10.3390/bioengineering9080392
work_keys_str_mv AT ghasemzadmahsa novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT hashemimahdieh novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT lavasanizohremiri novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT hosseinkhannazernikoo novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT bakhshandehhaleh novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT gramignoliroberto novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT keshavarzalikhanihani novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT najimimustapha novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT nikeghbaliansaman novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders
AT vosoughmassoud novelgenecorrectionbasedtherapeuticmodalitiesformonogenicliverdisorders