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Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

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Title: Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Authors: Costa-Verdera H; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.; Sorbonne University Paris and INSERM U974, 75013, Paris, France., Collaud F; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Riling CR; Spark Therapeutics, Philadelphia, PA, 19104, USA., Sellier P; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Nordin JML; Spark Therapeutics, Philadelphia, PA, 19104, USA., Preston GM; Spark Therapeutics, Philadelphia, PA, 19104, USA., Cagin U; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Fabregue J; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Barral S; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Moya-Nilges M; Pasteur Institute, 75015, Paris, France., Krijnse-Locker J; Pasteur Institute, 75015, Paris, France., van Wittenberghe L; Genethon, 91000, Evry, France., Daniele N; Genethon, 91000, Evry, France., Gjata B; Genethon, 91000, Evry, France., Cosette J; Genethon, 91000, Evry, France., Abad C; Université de Rouen Normandie-IRIB, 76183, Rouen, France., Simon-Sola M; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Charles S; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Li M; Spark Therapeutics, Philadelphia, PA, 19104, USA., Crosariol M; Spark Therapeutics, Philadelphia, PA, 19104, USA., Antrilli T; Spark Therapeutics, Philadelphia, PA, 19104, USA., Quinn WJ 3rd; Spark Therapeutics, Philadelphia, PA, 19104, USA., Gross DA; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Boyer O; Université de Rouen Normandie-IRIB, 76183, Rouen, France., Anguela XM; Spark Therapeutics, Philadelphia, PA, 19104, USA., Armour SM; Spark Therapeutics, Philadelphia, PA, 19104, USA., Colella P; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Ronzitti G; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Mingozzi F; Genethon, 91000, Evry, France. Federico.mingozzi@sparktx.com.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France. Federico.mingozzi@sparktx.com.; Sorbonne University Paris and INSERM U974, 75013, Paris, France. Federico.mingozzi@sparktx.com.; Spark Therapeutics, Philadelphia, PA, 19104, USA. Federico.mingozzi@sparktx.com.
Source: Nature communications [Nat Commun] 2021 Nov 04; Vol. 12 (1), pp. 6393. Date of Electronic Publication: 2021 Nov 04.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
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  Data: <searchLink fieldCode="AU" term="%22Costa-Verdera+H%22">Costa-Verdera H</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.; Sorbonne University Paris and INSERM U974, 75013, Paris, France.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Riling+CR%22">Riling CR</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Sellier+P%22">Sellier P</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Nordin+JML%22">Nordin JML</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Preston+GM%22">Preston GM</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Cagin+U%22">Cagin U</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Fabregue+J%22">Fabregue J</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Barral+S%22">Barral S</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Moya-Nilges+M%22">Moya-Nilges M</searchLink>; Pasteur Institute, 75015, Paris, France.<br /><searchLink fieldCode="AU" term="%22Krijnse-Locker+J%22">Krijnse-Locker J</searchLink>; Pasteur Institute, 75015, Paris, France.<br /><searchLink fieldCode="AU" term="%22van+Wittenberghe+L%22">van Wittenberghe L</searchLink>; Genethon, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Daniele+N%22">Daniele N</searchLink>; Genethon, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Gjata+B%22">Gjata B</searchLink>; Genethon, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Cosette+J%22">Cosette J</searchLink>; Genethon, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Abad+C%22">Abad C</searchLink>; Université de Rouen Normandie-IRIB, 76183, Rouen, France.<br /><searchLink fieldCode="AU" term="%22Simon-Sola+M%22">Simon-Sola M</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Charles+S%22">Charles S</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Li+M%22">Li M</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Crosariol+M%22">Crosariol M</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Antrilli+T%22">Antrilli T</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Quinn+WJ+3rd%22">Quinn WJ 3rd</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Gross+DA%22">Gross DA</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Boyer+O%22">Boyer O</searchLink>; Université de Rouen Normandie-IRIB, 76183, Rouen, France.<br /><searchLink fieldCode="AU" term="%22Anguela+XM%22">Anguela XM</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Armour+SM%22">Armour SM</searchLink>; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Colella+P%22">Colella P</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.<br /><searchLink fieldCode="AU" term="%22Mingozzi+F%22">Mingozzi F</searchLink>; Genethon, 91000, Evry, France. Federico.mingozzi@sparktx.com.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France. Federico.mingozzi@sparktx.com.; Sorbonne University Paris and INSERM U974, 75013, Paris, France. Federico.mingozzi@sparktx.com.; Spark Therapeutics, Philadelphia, PA, 19104, USA. Federico.mingozzi@sparktx.com.
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  Data: <searchLink fieldCode="JN" term="%22101528555%22">Nature communications</searchLink> [Nat Commun] 2021 Nov 04; Vol. 12 (1), pp. 6393. <i>Date of Electronic Publication: </i>2021 Nov 04.
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