Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
Pompe disease is currently treated with enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase (GAA). Here, the authors show hepatic-directed gene therapy with AAV vectors enhances GAA bioavailability compared with ERT, resulting in improved rescue of the disease phenotype in...
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Nature Portfolio
2021
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oai:doaj.org-article:ecdd7a66d5fe4b0e825ff6e616f835cf2021-11-08T11:11:07ZHepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates10.1038/s41467-021-26744-42041-1723https://doaj.org/article/ecdd7a66d5fe4b0e825ff6e616f835cf2021-11-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-26744-4https://doaj.org/toc/2041-1723Pompe disease is currently treated with enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase (GAA). Here, the authors show hepatic-directed gene therapy with AAV vectors enhances GAA bioavailability compared with ERT, resulting in improved rescue of the disease phenotype in mice and broad enzyme distribution in mice and non-human primates.Helena Costa-VerderaFanny CollaudChristopher R. RilingPauline SellierJayme M. L. NordinG. Michael PrestonUmut CaginJulien FabregueSimon BarralMaryse Moya-NilgesJacomina Krijnse-LockerLaetitia van WittenbergheNatalie DanieleBernard GjataJeremie CosetteCatalina AbadMarcelo Simon-SolaSeverine CharlesMathew LiMarco CrosariolTom AntrilliWilliam J. QuinnDavid A. GrossOlivier BoyerXavier M. AnguelaSean M. ArmourPasqualina ColellaGiuseppe RonzittiFederico MingozziNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-16 (2021) |
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DOAJ |
| collection |
DOAJ |
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EN |
| topic |
Science Q |
| spellingShingle |
Science Q Helena Costa-Verdera Fanny Collaud Christopher R. Riling Pauline Sellier Jayme M. L. Nordin G. Michael Preston Umut Cagin Julien Fabregue Simon Barral Maryse Moya-Nilges Jacomina Krijnse-Locker Laetitia van Wittenberghe Natalie Daniele Bernard Gjata Jeremie Cosette Catalina Abad Marcelo Simon-Sola Severine Charles Mathew Li Marco Crosariol Tom Antrilli William J. Quinn David A. Gross Olivier Boyer Xavier M. Anguela Sean M. Armour Pasqualina Colella Giuseppe Ronzitti Federico Mingozzi Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates |
| description |
Pompe disease is currently treated with enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase (GAA). Here, the authors show hepatic-directed gene therapy with AAV vectors enhances GAA bioavailability compared with ERT, resulting in improved rescue of the disease phenotype in mice and broad enzyme distribution in mice and non-human primates. |
| format |
article |
| author |
Helena Costa-Verdera Fanny Collaud Christopher R. Riling Pauline Sellier Jayme M. L. Nordin G. Michael Preston Umut Cagin Julien Fabregue Simon Barral Maryse Moya-Nilges Jacomina Krijnse-Locker Laetitia van Wittenberghe Natalie Daniele Bernard Gjata Jeremie Cosette Catalina Abad Marcelo Simon-Sola Severine Charles Mathew Li Marco Crosariol Tom Antrilli William J. Quinn David A. Gross Olivier Boyer Xavier M. Anguela Sean M. Armour Pasqualina Colella Giuseppe Ronzitti Federico Mingozzi |
| author_facet |
Helena Costa-Verdera Fanny Collaud Christopher R. Riling Pauline Sellier Jayme M. L. Nordin G. Michael Preston Umut Cagin Julien Fabregue Simon Barral Maryse Moya-Nilges Jacomina Krijnse-Locker Laetitia van Wittenberghe Natalie Daniele Bernard Gjata Jeremie Cosette Catalina Abad Marcelo Simon-Sola Severine Charles Mathew Li Marco Crosariol Tom Antrilli William J. Quinn David A. Gross Olivier Boyer Xavier M. Anguela Sean M. Armour Pasqualina Colella Giuseppe Ronzitti Federico Mingozzi |
| author_sort |
Helena Costa-Verdera |
| title |
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates |
| title_short |
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates |
| title_full |
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates |
| title_fullStr |
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates |
| title_full_unstemmed |
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates |
| title_sort |
hepatic expression of gaa results in enhanced enzyme bioavailability in mice and non-human primates |
| publisher |
Nature Portfolio |
| publishDate |
2021 |
| url |
https://doaj.org/article/ecdd7a66d5fe4b0e825ff6e616f835cf |
| work_keys_str_mv |
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