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Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model.

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Title: Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model.
Authors: Milenkovic I; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.; Department of Neurology, Medical University of Vienna, Vienna, Austria., Blumenreich S; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Hochfelder A; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Azulay A; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Biton IE; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Zerbib M; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Oren R; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Tsoory M; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Joseph T; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Fleishman SJ; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Futerman AH; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel. tony.futerman@weizmann.ac.il.
Source: Gene therapy [Gene Ther] 2024 Sep; Vol. 31 (9-10), pp. 439-444. Date of Electronic Publication: 2024 Aug 15.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model.
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  Data: <searchLink fieldCode="AU" term="%22Milenkovic+I%22">Milenkovic I</searchLink>; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.; Department of Neurology, Medical University of Vienna, Vienna, Austria.<br /><searchLink fieldCode="AU" term="%22Blumenreich+S%22">Blumenreich S</searchLink>; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Hochfelder+A%22">Hochfelder A</searchLink>; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Azulay+A%22">Azulay A</searchLink>; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Biton+IE%22">Biton IE</searchLink>; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Zerbib+M%22">Zerbib M</searchLink>; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Oren+R%22">Oren R</searchLink>; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Tsoory+M%22">Tsoory M</searchLink>; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Joseph+T%22">Joseph T</searchLink>; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Fleishman+SJ%22">Fleishman SJ</searchLink>; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.<br /><searchLink fieldCode="AU" term="%22Futerman+AH%22">Futerman AH</searchLink>; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel. tony.futerman@weizmann.ac.il.
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  Data: <searchLink fieldCode="JN" term="%229421525%22">Gene therapy</searchLink> [Gene Ther] 2024 Sep; Vol. 31 (9-10), pp. 439-444. <i>Date of Electronic Publication: </i>2024 Aug 15.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Publishing+Group%22">Nature Publishing Group </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>9421525 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1476-5462 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209697128%22">09697128 </searchLink><i>NLM ISO Abbreviation: </i>Gene Ther <i>Subsets: </i>MEDLINE
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