Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease
Pompe disease is caused by the deficiency of lysosomal acid α-glucosidase (GAA) leading to progressive myopathy. Enzyme replacement therapy (ERT) with recombinant human (rh) GAA has limitations, including inefficient uptake of rhGAA in skeletal muscle linked to low cation-independent mannose-6-phosp...
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| Veröffentlicht in: | Molecular genetics and metabolism 2020-02, Vol.129 (2), p.73-79 |
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| creator | Han, Sang-oh Haynes, Alexina C. Li, Songtao Abraham, Dennis M. Kishnani, Priya S. Steet, Richard Koeberl, Dwight D. |
| description | Pompe disease is caused by the deficiency of lysosomal acid α-glucosidase (GAA) leading to progressive myopathy. Enzyme replacement therapy (ERT) with recombinant human (rh) GAA has limitations, including inefficient uptake of rhGAA in skeletal muscle linked to low cation-independent mannose-6-phosphate receptor (CI-MPR) expression.
To test the hypothesis that antihypertensive agents causing muscle hypertrophy by increasing insulin-like growth factor 1 expression can increase CI-MPR-mediated uptake of recombinant enzyme with therapeutic effects in skeletal muscle.
Three such agents were evaluated in mice with Pompe disease (carvedilol, losartan, and propranolol), either with or without concurrent ERT.
Carvedilol, a selective β-blocker, increased muscle strength but reduced biochemical correction from ERT. Administration of drugs alone had minimal effect, with the exception of losartan that increased glycogen storage and mortality either by itself or in combination with ERT.
The β-blocker carvedilol had beneficial effects during ERT in mice with Pompe disease, in comparison with propranolol or losartan. Caution is warranted when prescribing antihypertensive drugs in Pompe disease. |
| doi_str_mv | 10.1016/j.ymgme.2019.10.005 |
| format | Article |
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To test the hypothesis that antihypertensive agents causing muscle hypertrophy by increasing insulin-like growth factor 1 expression can increase CI-MPR-mediated uptake of recombinant enzyme with therapeutic effects in skeletal muscle.
Three such agents were evaluated in mice with Pompe disease (carvedilol, losartan, and propranolol), either with or without concurrent ERT.
Carvedilol, a selective β-blocker, increased muscle strength but reduced biochemical correction from ERT. Administration of drugs alone had minimal effect, with the exception of losartan that increased glycogen storage and mortality either by itself or in combination with ERT.
The β-blocker carvedilol had beneficial effects during ERT in mice with Pompe disease, in comparison with propranolol or losartan. Caution is warranted when prescribing antihypertensive drugs in Pompe disease.</description><identifier>ISSN: 1096-7192</identifier><identifier>EISSN: 1096-7206</identifier><identifier>DOI: 10.1016/j.ymgme.2019.10.005</identifier><identifier>PMID: 31645300</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Acid alpha-glucosidase ; Acid maltase ; alpha-Glucosidases - genetics ; Animals ; Antihypertensive Agents - therapeutic use ; Disease Models, Animal ; Drug Therapy, Combination ; Enzyme Replacement Therapy ; Female ; Glycogen storage disease type II ; Glycogen Storage Disease Type II - drug therapy ; Insulin-Like Growth Factor I - genetics ; Male ; Mannose-6-phosphate receptor ; Mice ; Mice, Knockout ; Muscle, Skeletal - drug effects ; Muscle, Skeletal - pathology</subject><ispartof>Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.73-79</ispartof><rights>2019</rights><rights>Copyright © 2019. Published by Elsevier Inc.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c459t-c23744efa68784ef158975672a22e2ce4a3c91c10fea98d5b9e6a4cbce0a1ff83</citedby><cites>FETCH-LOGICAL-c459t-c23744efa68784ef158975672a22e2ce4a3c91c10fea98d5b9e6a4cbce0a1ff83</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.ymgme.2019.10.005$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>230,314,780,784,885,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/31645300$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Han, Sang-oh</creatorcontrib><creatorcontrib>Haynes, Alexina C.</creatorcontrib><creatorcontrib>Li, Songtao</creatorcontrib><creatorcontrib>Abraham, Dennis M.</creatorcontrib><creatorcontrib>Kishnani, Priya S.</creatorcontrib><creatorcontrib>Steet, Richard</creatorcontrib><creatorcontrib>Koeberl, Dwight D.</creatorcontrib><title>Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease</title><title>Molecular genetics and metabolism</title><addtitle>Mol Genet Metab</addtitle><description>Pompe disease is caused by the deficiency of lysosomal acid α-glucosidase (GAA) leading to progressive myopathy. Enzyme replacement therapy (ERT) with recombinant human (rh) GAA has limitations, including inefficient uptake of rhGAA in skeletal muscle linked to low cation-independent mannose-6-phosphate receptor (CI-MPR) expression.
To test the hypothesis that antihypertensive agents causing muscle hypertrophy by increasing insulin-like growth factor 1 expression can increase CI-MPR-mediated uptake of recombinant enzyme with therapeutic effects in skeletal muscle.
Three such agents were evaluated in mice with Pompe disease (carvedilol, losartan, and propranolol), either with or without concurrent ERT.
Carvedilol, a selective β-blocker, increased muscle strength but reduced biochemical correction from ERT. Administration of drugs alone had minimal effect, with the exception of losartan that increased glycogen storage and mortality either by itself or in combination with ERT.
The β-blocker carvedilol had beneficial effects during ERT in mice with Pompe disease, in comparison with propranolol or losartan. Caution is warranted when prescribing antihypertensive drugs in Pompe disease.</description><subject>Acid alpha-glucosidase</subject><subject>Acid maltase</subject><subject>alpha-Glucosidases - genetics</subject><subject>Animals</subject><subject>Antihypertensive Agents - therapeutic use</subject><subject>Disease Models, Animal</subject><subject>Drug Therapy, Combination</subject><subject>Enzyme Replacement Therapy</subject><subject>Female</subject><subject>Glycogen storage disease type II</subject><subject>Glycogen Storage Disease Type II - drug therapy</subject><subject>Insulin-Like Growth Factor I - genetics</subject><subject>Male</subject><subject>Mannose-6-phosphate receptor</subject><subject>Mice</subject><subject>Mice, Knockout</subject><subject>Muscle, Skeletal - drug effects</subject><subject>Muscle, Skeletal - pathology</subject><issn>1096-7192</issn><issn>1096-7206</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kU1vFDEMhiMEoqXwC5DQHLns4mQ-cwAJVeVDqgQHOEfejGc3q0kyJJlFw68ny7QVXDjZsh-_tvwy9pLDlgNv3hy3i91b2grgMle2APUjdslBNptWQPP4PudSXLBnMR4BOK9l9ZRdlLyp6hLgkrmbE44zJuNd4YcCXTKHZaKQyEVzoqIP8z4WxhXa251xK_jTpENB7tdiqQg0jajJkktFOlDAaTnj1mhaua_eTlnHRMJIz9mTAcdIL-7iFfv-4ebb9afN7ZePn6_f3250Vcu00aJsq4oGbLq2y5HXnWzrphUoBAlNFZZacs1hIJRdX-8kNVjpnSZAPgxdecXerbrTvLPU63xdwFFNwVgMi_Jo1L8dZw5q70-qBRACZBZ4fScQ_I-ZYlLWRE3jiI78HJUooasFL3mV0XJFdfAxBhoe1nBQZ6fUUf1xSp2dOhezU3nq1d8XPszcW5OBtytA-U8nQ0FFbchp6k0gnVTvzX8X_AaaM6n1</recordid><startdate>20200201</startdate><enddate>20200201</enddate><creator>Han, Sang-oh</creator><creator>Haynes, Alexina C.</creator><creator>Li, Songtao</creator><creator>Abraham, Dennis M.</creator><creator>Kishnani, Priya S.</creator><creator>Steet, Richard</creator><creator>Koeberl, Dwight D.</creator><general>Elsevier Inc</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>20200201</creationdate><title>Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease</title><author>Han, Sang-oh ; Haynes, Alexina C. ; Li, Songtao ; Abraham, Dennis M. ; Kishnani, Priya S. ; Steet, Richard ; Koeberl, Dwight D.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c459t-c23744efa68784ef158975672a22e2ce4a3c91c10fea98d5b9e6a4cbce0a1ff83</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>Acid alpha-glucosidase</topic><topic>Acid maltase</topic><topic>alpha-Glucosidases - genetics</topic><topic>Animals</topic><topic>Antihypertensive Agents - therapeutic use</topic><topic>Disease Models, Animal</topic><topic>Drug Therapy, Combination</topic><topic>Enzyme Replacement Therapy</topic><topic>Female</topic><topic>Glycogen storage disease type II</topic><topic>Glycogen Storage Disease Type II - drug therapy</topic><topic>Insulin-Like Growth Factor I - genetics</topic><topic>Male</topic><topic>Mannose-6-phosphate receptor</topic><topic>Mice</topic><topic>Mice, Knockout</topic><topic>Muscle, Skeletal - drug effects</topic><topic>Muscle, Skeletal - pathology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Han, Sang-oh</creatorcontrib><creatorcontrib>Haynes, Alexina C.</creatorcontrib><creatorcontrib>Li, Songtao</creatorcontrib><creatorcontrib>Abraham, Dennis M.</creatorcontrib><creatorcontrib>Kishnani, Priya S.</creatorcontrib><creatorcontrib>Steet, Richard</creatorcontrib><creatorcontrib>Koeberl, Dwight D.</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Molecular genetics and metabolism</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Han, Sang-oh</au><au>Haynes, Alexina C.</au><au>Li, Songtao</au><au>Abraham, Dennis M.</au><au>Kishnani, Priya S.</au><au>Steet, Richard</au><au>Koeberl, Dwight D.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease</atitle><jtitle>Molecular genetics and metabolism</jtitle><addtitle>Mol Genet Metab</addtitle><date>2020-02-01</date><risdate>2020</risdate><volume>129</volume><issue>2</issue><spage>73</spage><epage>79</epage><pages>73-79</pages><issn>1096-7192</issn><eissn>1096-7206</eissn><abstract>Pompe disease is caused by the deficiency of lysosomal acid α-glucosidase (GAA) leading to progressive myopathy. Enzyme replacement therapy (ERT) with recombinant human (rh) GAA has limitations, including inefficient uptake of rhGAA in skeletal muscle linked to low cation-independent mannose-6-phosphate receptor (CI-MPR) expression.
To test the hypothesis that antihypertensive agents causing muscle hypertrophy by increasing insulin-like growth factor 1 expression can increase CI-MPR-mediated uptake of recombinant enzyme with therapeutic effects in skeletal muscle.
Three such agents were evaluated in mice with Pompe disease (carvedilol, losartan, and propranolol), either with or without concurrent ERT.
Carvedilol, a selective β-blocker, increased muscle strength but reduced biochemical correction from ERT. Administration of drugs alone had minimal effect, with the exception of losartan that increased glycogen storage and mortality either by itself or in combination with ERT.
The β-blocker carvedilol had beneficial effects during ERT in mice with Pompe disease, in comparison with propranolol or losartan. Caution is warranted when prescribing antihypertensive drugs in Pompe disease.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>31645300</pmid><doi>10.1016/j.ymgme.2019.10.005</doi><tpages>7</tpages><oa>free_for_read</oa></addata></record> |
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| source | MEDLINE; ScienceDirect Journals (5 years ago - present) |
| subjects | Acid alpha-glucosidase Acid maltase alpha-Glucosidases - genetics Animals Antihypertensive Agents - therapeutic use Disease Models, Animal Drug Therapy, Combination Enzyme Replacement Therapy Female Glycogen storage disease type II Glycogen Storage Disease Type II - drug therapy Insulin-Like Growth Factor I - genetics Male Mannose-6-phosphate receptor Mice Mice, Knockout Muscle, Skeletal - drug effects Muscle, Skeletal - pathology |
| title | Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease |
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