Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria

Porphobilinogen deaminase (PBGD) haploinsufficiency (acute intermittent porphyria, AIP) is characterized by neurovisceral attacks associated with high production, accumulation and urinary excretion of heme precursors, δ-aminolevulinic acid (ALA) and porphobilinogen (PBG). The estimated clinical pene...

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Autores: Solares, Isabel, Jericó, Daniel, Córdoba, Karol Marcela, Morales Conejo, Montserrat, Ena, Javier, Enríquez De Salamanca Lorente, Rafael, Fontanellas, Antonio
Tipo de recurso: artículo
Fecha de publicación:2022
País:España
Institución:Universidad Complutense de Madrid (UCM)
Repositorio:Docta Complutense
Idioma:inglés
OAI Identifier:oai:docta.ucm.es:20.500.14352/72333
Acceso en línea:https://hdl.handle.net/20.500.14352/72333
Access Level:acceso abierto
Palabra clave:616.3
Hepatic porphyrias
Acute intermittent porphyria
Fasting
Glucose homeostasis
Insulin resistance
Mitochondrial function and biogenesis
Medicina
Endocrinología
Gastroenterología y hepatología
Medicina interna
32 Ciencias Médicas
3205.02 Endocrinología
3205.03 Gastroenterología
3205 Medicina Interna
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oai_identifier_str oai:docta.ucm.es:20.500.14352/72333
network_acronym_str ES
network_name_str España
repository_id_str
spelling Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent PorphyriaSolares, IsabelJericó, DanielCórdoba, Karol MarcelaMorales Conejo, MontserratEna, JavierEnríquez De Salamanca Lorente, RafaelFontanellas, Antonio616.3Hepatic porphyriasAcute intermittent porphyriaFastingGlucose homeostasisInsulin resistanceMitochondrial function and biogenesisMedicinaEndocrinologíaGastroenterología y hepatologíaMedicina interna32 Ciencias Médicas3205.02 Endocrinología3205.03 Gastroenterología3205 Medicina InternaPorphobilinogen deaminase (PBGD) haploinsufficiency (acute intermittent porphyria, AIP) is characterized by neurovisceral attacks associated with high production, accumulation and urinary excretion of heme precursors, δ-aminolevulinic acid (ALA) and porphobilinogen (PBG). The estimated clinical penetrance for AIP is extremely low (<1%), therefore it is likely that other factors may play an important role in the predisposition to developing attacks. Fasting is a known triggering factor. Given the increased prevalence of insulin resistance in patients and the large urinary loss of succinyl-CoA to produce ALA and PBG, we explore the impact of reduced availability of energy metabolites in the severity of AIP pathophysiology. Classic studies found clinical improvement in patients affected by AIP associated with the administration of glucose and concomitant insulin secretion, or after hyperinsulinemia associated with diabetes. Molecular studies have confirmed that glucose and insulin administration induces a repressive effect on hepatic ALA Synthase, the first and regulatory step of the heme pathway. More recently, the insulin-mimicking α-lipoic acid has been shown to improve glucose metabolism and mitochondrial dysfunction in a hepatocyte cell line transfected with interfering RNA targeting PBGD. In AIP mice, preventive treatment with an experimental fusion protein of insulin and apolipoprotein A-I improved the disease by promoting fat mobilization in adipose tissue, increasing the metabolite bioavailability for the TCA cycle and inducing mitochondrial biogenesis in the liver. In this review, we analyze the possible mechanisms underlying abnormal hepatocellular carbohydrate homeostasis in AIP.MDPIUniversidad Complutense de Madrid20222022-12-2020222022-12-20journal articlehttp://purl.org/coar/resource_type/c_6501info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/20.500.14352/72333reponame:Docta Complutenseinstname:Universidad Complutense de Madrid (UCM)InglésengInstituto de Salud Carlos III http://dx.doi.org/10.13039/501100004587 Plan Estatal de Investigación Científica y Técnica y de Innovación 2013-2016 (ISCIII) PI18%2F00860 ENSAYO PRECLINICO CON ARN MENSAJERO PARA EL TRATAMIENTO ETIOLOGICO DE LA PORFIRIA AGUDA INTERMITENTE. EFICACIA Y SEGURIDAD EN PRIMATES NO HUMANOS Y EN UN NUEVO MODELO DE PORFIRIA HEPATICA EN CONEJO .Instituto de Salud Carlos III http://dx.doi.org/10.13039/501100004587 Plan Estatal de Investigación Científica y Técnica y de Innovación 2017-2020 (ISCIII) PI21%2F00546 EVALUACION DE MEDICAMENTOS INNOVADORES BASADOS EN ARN EN LA CORRECCION DE LAS VIAS METABÓLICAS DE HEMO, GLICOLIPIDOS Y HOMOCISTEINA EN PACIENTES CON PORFIRIA AGUDA INTERMITENTE Y MODELOS EXPERIMENTALES DE RATÓN Y PRIMATES-NO-HUMANOS.open accesshttp://purl.org/coar/access_right/c_abf2Atribución 3.0 Españahttps://creativecommons.org/licenses/by/3.0/es/info:eu-repo/semantics/openAccessoai:docta.ucm.es:20.500.14352/723332026-06-02T12:44:21Z
dc.title.none.fl_str_mv Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
title Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
spellingShingle Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
Solares, Isabel
616.3
Hepatic porphyrias
Acute intermittent porphyria
Fasting
Glucose homeostasis
Insulin resistance
Mitochondrial function and biogenesis
Medicina
Endocrinología
Gastroenterología y hepatología
Medicina interna
32 Ciencias Médicas
3205.02 Endocrinología
3205.03 Gastroenterología
3205 Medicina Interna
title_short Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
title_full Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
title_fullStr Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
title_full_unstemmed Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
title_sort Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
dc.creator.none.fl_str_mv Solares, Isabel
Jericó, Daniel
Córdoba, Karol Marcela
Morales Conejo, Montserrat
Ena, Javier
Enríquez De Salamanca Lorente, Rafael
Fontanellas, Antonio
author Solares, Isabel
author_facet Solares, Isabel
Jericó, Daniel
Córdoba, Karol Marcela
Morales Conejo, Montserrat
Ena, Javier
Enríquez De Salamanca Lorente, Rafael
Fontanellas, Antonio
author_role author
author2 Jericó, Daniel
Córdoba, Karol Marcela
Morales Conejo, Montserrat
Ena, Javier
Enríquez De Salamanca Lorente, Rafael
Fontanellas, Antonio
author2_role author
author
author
author
author
author
dc.contributor.none.fl_str_mv Universidad Complutense de Madrid
dc.subject.none.fl_str_mv 616.3
Hepatic porphyrias
Acute intermittent porphyria
Fasting
Glucose homeostasis
Insulin resistance
Mitochondrial function and biogenesis
Medicina
Endocrinología
Gastroenterología y hepatología
Medicina interna
32 Ciencias Médicas
3205.02 Endocrinología
3205.03 Gastroenterología
3205 Medicina Interna
topic 616.3
Hepatic porphyrias
Acute intermittent porphyria
Fasting
Glucose homeostasis
Insulin resistance
Mitochondrial function and biogenesis
Medicina
Endocrinología
Gastroenterología y hepatología
Medicina interna
32 Ciencias Médicas
3205.02 Endocrinología
3205.03 Gastroenterología
3205 Medicina Interna
description Porphobilinogen deaminase (PBGD) haploinsufficiency (acute intermittent porphyria, AIP) is characterized by neurovisceral attacks associated with high production, accumulation and urinary excretion of heme precursors, δ-aminolevulinic acid (ALA) and porphobilinogen (PBG). The estimated clinical penetrance for AIP is extremely low (<1%), therefore it is likely that other factors may play an important role in the predisposition to developing attacks. Fasting is a known triggering factor. Given the increased prevalence of insulin resistance in patients and the large urinary loss of succinyl-CoA to produce ALA and PBG, we explore the impact of reduced availability of energy metabolites in the severity of AIP pathophysiology. Classic studies found clinical improvement in patients affected by AIP associated with the administration of glucose and concomitant insulin secretion, or after hyperinsulinemia associated with diabetes. Molecular studies have confirmed that glucose and insulin administration induces a repressive effect on hepatic ALA Synthase, the first and regulatory step of the heme pathway. More recently, the insulin-mimicking α-lipoic acid has been shown to improve glucose metabolism and mitochondrial dysfunction in a hepatocyte cell line transfected with interfering RNA targeting PBGD. In AIP mice, preventive treatment with an experimental fusion protein of insulin and apolipoprotein A-I improved the disease by promoting fat mobilization in adipose tissue, increasing the metabolite bioavailability for the TCA cycle and inducing mitochondrial biogenesis in the liver. In this review, we analyze the possible mechanisms underlying abnormal hepatocellular carbohydrate homeostasis in AIP.
publishDate 2022
dc.date.none.fl_str_mv 2022
2022-12-20
2022
2022-12-20
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/20.500.14352/72333
url https://hdl.handle.net/20.500.14352/72333
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.relation.none.fl_str_mv Instituto de Salud Carlos III http://dx.doi.org/10.13039/501100004587 Plan Estatal de Investigación Científica y Técnica y de Innovación 2013-2016 (ISCIII) PI18%2F00860 ENSAYO PRECLINICO CON ARN MENSAJERO PARA EL TRATAMIENTO ETIOLOGICO DE LA PORFIRIA AGUDA INTERMITENTE. EFICACIA Y SEGURIDAD EN PRIMATES NO HUMANOS Y EN UN NUEVO MODELO DE PORFIRIA HEPATICA EN CONEJO .
Instituto de Salud Carlos III http://dx.doi.org/10.13039/501100004587 Plan Estatal de Investigación Científica y Técnica y de Innovación 2017-2020 (ISCIII) PI21%2F00546 EVALUACION DE MEDICAMENTOS INNOVADORES BASADOS EN ARN EN LA CORRECCION DE LAS VIAS METABÓLICAS DE HEMO, GLICOLIPIDOS Y HOMOCISTEINA EN PACIENTES CON PORFIRIA AGUDA INTERMITENTE Y MODELOS EXPERIMENTALES DE RATÓN Y PRIMATES-NO-HUMANOS.
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Atribución 3.0 España
https://creativecommons.org/licenses/by/3.0/es/
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
Atribución 3.0 España
https://creativecommons.org/licenses/by/3.0/es/
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:Docta Complutense
instname:Universidad Complutense de Madrid (UCM)
instname_str Universidad Complutense de Madrid (UCM)
reponame_str Docta Complutense
collection Docta Complutense
repository.name.fl_str_mv
repository.mail.fl_str_mv
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