Advanced therapies for the treatment of hemophilia: future perspectives

Monogenic diseases are ideal candidates for treatment by the emerging advanced therapies, which are capable of correcting alterations in protein expression that result from genetic mutation. In hemophilia A and B such alterations affect the activity of coagulation factors VIII and IX, respectively,...

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Detalles Bibliográficos
Autores: Liras, Antonio, Segovia, Cristina, Gaban, Aline S.
Tipo de recurso: artículo
Fecha de publicación:2012
País:España
Institución:Universidad Complutense de Madrid (UCM)
Repositorio:Docta Complutense
Idioma:inglés
OAI Identifier:oai:docta.ucm.es:20.500.14352/44628
Acceso en línea:https://hdl.handle.net/20.500.14352/44628
Access Level:acceso abierto
Palabra clave:616.151.5
Advanced therapies
Gene therapy
Cell therapy
Hemophilia A
Hemophilia B
Farmacología (Medicina)
Genética médica
Hematología
2410.07 Genética Humana
3205.04 Hematología
id ES_707992a6bcfcb0ff00a836c0781e9ef9
oai_identifier_str oai:docta.ucm.es:20.500.14352/44628
network_acronym_str ES
network_name_str España
spelling Advanced therapies for the treatment of hemophilia: future perspectives Liras, Antonio Segovia, Cristina Gaban, Aline S. 616.151.5 Advanced therapies Gene therapy Cell therapy Hemophilia A Hemophilia B Farmacología (Medicina) Genética médica Hematología 2410.07 Genética Humana 3205.04 Hematología Monogenic diseases are ideal candidates for treatment by the emerging advanced therapies, which are capable of correcting alterations in protein expression that result from genetic mutation. In hemophilia A and B such alterations affect the activity of coagulation factors VIII and IX, respectively, and are responsible for the development of the disease. Advanced therapies may involve the replacement of a deficient gene by a healthy gene so that it generates a certain functional,structural or transport protein (gene therapy); the incorporation of a full array of healthy genes and proteins through perfusion or transplantation of healthy cells (cell therapy); or tissue transplantation and formation of healthy organs (tissue engineering). For their part, induced pluripotent stem cells have recently been shown to also play a significant role in the fields of cell therapy and tissue engineering.Hemophilia is optimally suited for advanced therapies owing to the fact that, as a monogenic condition, it does not require very high expression levels of a coagulation factor to reach moderate disease status. As a result, significant progress has been possible with respect to these kinds of strategies, especially in the fields of gene therapy (by using viral and non-viral vectors) and cell therapy (by means of several types of target cells). Thus, although still considered a rare disorder, hemophilia is now recognized as a condition amenable to gene therapy, which can be administered in the form of lentiviral and adeno-associated vectors applied to adult stem cells, autologous fibroblasts, platelets and hematopoietic stem cells; by means of non-viral vectors; or through the repair of mutations by chimeric oligonucleotides. In hemophilia, cell therapy approaches have been based mainly on transplantation of healthy cells (adult stem cells or induced pluripotent cell-derived progenitor cells)in order to restore alterations in coagulation factor expression. BioMed Central https://hdl.handle.net/20.500.14352/44628
title Advanced therapies for the treatment of hemophilia: future perspectives
spellingShingle Advanced therapies for the treatment of hemophilia: future perspectives
Liras, Antonio
616.151.5
Advanced therapies
Gene therapy
Cell therapy
Hemophilia A
Hemophilia B
Farmacología (Medicina)
Genética médica
Hematología
2410.07 Genética Humana
3205.04 Hematología
title_short Advanced therapies for the treatment of hemophilia: future perspectives
title_full Advanced therapies for the treatment of hemophilia: future perspectives
title_fullStr Advanced therapies for the treatment of hemophilia: future perspectives
title_full_unstemmed Advanced therapies for the treatment of hemophilia: future perspectives
title_sort Advanced therapies for the treatment of hemophilia: future perspectives
author Liras, Antonio
author_facet Liras, Antonio
Segovia, Cristina
Gaban, Aline S.
author_role author
author2 Segovia, Cristina
Gaban, Aline S.
author2_role author
author
topic 616.151.5
Advanced therapies
Gene therapy
Cell therapy
Hemophilia A
Hemophilia B
Farmacología (Medicina)
Genética médica
Hematología
2410.07 Genética Humana
3205.04 Hematología
topic_facet 616.151.5
Advanced therapies
Gene therapy
Cell therapy
Hemophilia A
Hemophilia B
Farmacología (Medicina)
Genética médica
Hematología
2410.07 Genética Humana
3205.04 Hematología
description Monogenic diseases are ideal candidates for treatment by the emerging advanced therapies, which are capable of correcting alterations in protein expression that result from genetic mutation. In hemophilia A and B such alterations affect the activity of coagulation factors VIII and IX, respectively, and are responsible for the development of the disease. Advanced therapies may involve the replacement of a deficient gene by a healthy gene so that it generates a certain functional,structural or transport protein (gene therapy); the incorporation of a full array of healthy genes and proteins through perfusion or transplantation of healthy cells (cell therapy); or tissue transplantation and formation of healthy organs (tissue engineering). For their part, induced pluripotent stem cells have recently been shown to also play a significant role in the fields of cell therapy and tissue engineering.Hemophilia is optimally suited for advanced therapies owing to the fact that, as a monogenic condition, it does not require very high expression levels of a coagulation factor to reach moderate disease status. As a result, significant progress has been possible with respect to these kinds of strategies, especially in the fields of gene therapy (by using viral and non-viral vectors) and cell therapy (by means of several types of target cells). Thus, although still considered a rare disorder, hemophilia is now recognized as a condition amenable to gene therapy, which can be administered in the form of lentiviral and adeno-associated vectors applied to adult stem cells, autologous fibroblasts, platelets and hematopoietic stem cells; by means of non-viral vectors; or through the repair of mutations by chimeric oligonucleotides. In hemophilia, cell therapy approaches have been based mainly on transplantation of healthy cells (adult stem cells or induced pluripotent cell-derived progenitor cells)in order to restore alterations in coagulation factor expression.
publishDate 2012
format article
url https://hdl.handle.net/20.500.14352/44628
language eng
eu_rights_str_mv openAccess
publisher BioMed Central
institution Universidad Complutense de Madrid (UCM)
collection Docta Complutense
reponame_str Docta Complutense
instname_str Universidad Complutense de Madrid (UCM)
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publishDateSort 2012
author_browse Gaban, Aline S.
Liras, Antonio
Segovia, Cristina
publisherStr BioMed Central
score 6,924472

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