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. |
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| 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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| ISSN: | 2041-1723 |
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| DOI: | 10.1038/s41467-021-26744-4 |
