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Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model.

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Title: Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model.
Authors: Cocozza G; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy., D'Andrea T; IRCCS Neuromed, Pozzilli, Italy., Di Rocco M; Department of Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy., Busdraghi LM; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy., Chilà G; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy., Lin X; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy., Lanza E; D-tails, Rome, Italy., Collaud F; Université Paris-Saclay, University Evry, Integrare Research Unit UMR_S951, Inserm, Genethon, Evry, 91000, France.; Genethon, Evry, 91000, France., Follo FC; Department of Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy., Buccheri C; National Research Council (Institute of Biochemistry and Cell Biology), Campus International Development (European Mouse Mutant Archive- INFRAFRONTIER), Monterotondo Scalo, Rome, Italy., Folli V; D-tails, Rome, Italy., Ronzitti G; Université Paris-Saclay, University Evry, Integrare Research Unit UMR_S951, Inserm, Genethon, Evry, 91000, France.; Genethon, Evry, 91000, France., Scavizzi F; National Research Council (Institute of Biochemistry and Cell Biology), Campus International Development (European Mouse Mutant Archive- INFRAFRONTIER), Monterotondo Scalo, Rome, Italy., Raspa M; National Research Council (Institute of Biochemistry and Cell Biology), Campus International Development (European Mouse Mutant Archive- INFRAFRONTIER), Monterotondo Scalo, Rome, Italy., Leuzzi V; Department of Human Neuroscience, Sapienza University of Rome, Rome, Italy., Katanaev VL; Department of Cell Physiology and Metabolism, Faculty of Medicine, Translational Research Center in Oncohaematology, University of Geneva, Geneva, 1211, Switzerland.; Qatar Biomedical Research Institute, College of Health and Life Sciences, Hamad Bin Khalifa University, P.O. Box 34110, Doha, Qatar., Galosi S; Department of Human Neuroscience, Sapienza University of Rome, Rome, Italy., Fucile S; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy.; IRCCS Neuromed, Pozzilli, Italy., Martinelli S; Department of Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy., D'Alessandro G; IRCCS Neuromed, Pozzilli, Italy. giuseppina.dalessandro@uniroma1.it.; Laboratory affiliated to Istituto Pasteur Rome, Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy. giuseppina.dalessandro@uniroma1.it., Limatola C; IRCCS Neuromed, Pozzilli, Italy. cristina.limatola@uniroma1.it.; Laboratory affiliated to Istituto Pasteur Rome, Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy. cristina.limatola@uniroma1.it.
Source: Acta neuropathologica communications [Acta Neuropathol Commun] 2026 Jul 21. Date of Electronic Publication: 2026 Jul 21.
Publication Type: Journal Article
Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101610673 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2051-5960 (Electronic) Linking ISSN: 20515960 NLM ISO Abbreviation: Acta Neuropathol Commun Subsets: MEDLINE
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  Data: Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model.
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  Data: <searchLink fieldCode="AU" term="%22Cocozza+G%22">Cocozza G</searchLink>; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22D'Andrea+T%22">D'Andrea T</searchLink>; IRCCS Neuromed, Pozzilli, Italy.<br /><searchLink fieldCode="AU" term="%22Di+Rocco+M%22">Di Rocco M</searchLink>; Department of Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Busdraghi+LM%22">Busdraghi LM</searchLink>; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Chilà+G%22">Chilà G</searchLink>; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Lin+X%22">Lin X</searchLink>; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Lanza+E%22">Lanza E</searchLink>; D-tails, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Université Paris-Saclay, University Evry, Integrare Research Unit UMR_S951, Inserm, Genethon, Evry, 91000, France.; Genethon, Evry, 91000, France.<br /><searchLink fieldCode="AU" term="%22Follo+FC%22">Follo FC</searchLink>; Department of Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Buccheri+C%22">Buccheri C</searchLink>; National Research Council (Institute of Biochemistry and Cell Biology), Campus International Development (European Mouse Mutant Archive- INFRAFRONTIER), Monterotondo Scalo, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Folli+V%22">Folli V</searchLink>; D-tails, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Université Paris-Saclay, University Evry, Integrare Research Unit UMR_S951, Inserm, Genethon, Evry, 91000, France.; Genethon, Evry, 91000, France.<br /><searchLink fieldCode="AU" term="%22Scavizzi+F%22">Scavizzi F</searchLink>; National Research Council (Institute of Biochemistry and Cell Biology), Campus International Development (European Mouse Mutant Archive- INFRAFRONTIER), Monterotondo Scalo, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Raspa+M%22">Raspa M</searchLink>; National Research Council (Institute of Biochemistry and Cell Biology), Campus International Development (European Mouse Mutant Archive- INFRAFRONTIER), Monterotondo Scalo, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Leuzzi+V%22">Leuzzi V</searchLink>; Department of Human Neuroscience, Sapienza University of Rome, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Katanaev+VL%22">Katanaev VL</searchLink>; Department of Cell Physiology and Metabolism, Faculty of Medicine, Translational Research Center in Oncohaematology, University of Geneva, Geneva, 1211, Switzerland.; Qatar Biomedical Research Institute, College of Health and Life Sciences, Hamad Bin Khalifa University, P.O. Box 34110, Doha, Qatar.<br /><searchLink fieldCode="AU" term="%22Galosi+S%22">Galosi S</searchLink>; Department of Human Neuroscience, Sapienza University of Rome, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Fucile+S%22">Fucile S</searchLink>; Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy.; IRCCS Neuromed, Pozzilli, Italy.<br /><searchLink fieldCode="AU" term="%22Martinelli+S%22">Martinelli S</searchLink>; Department of Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22D'Alessandro+G%22">D'Alessandro G</searchLink>; IRCCS Neuromed, Pozzilli, Italy. giuseppina.dalessandro@uniroma1.it.; Laboratory affiliated to Istituto Pasteur Rome, Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy. giuseppina.dalessandro@uniroma1.it.<br /><searchLink fieldCode="AU" term="%22Limatola+C%22">Limatola C</searchLink>; IRCCS Neuromed, Pozzilli, Italy. cristina.limatola@uniroma1.it.; Laboratory affiliated to Istituto Pasteur Rome, Department of Physiology and Pharmacology, Sapienza University of Rome, Rome, Italy. cristina.limatola@uniroma1.it.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22BioMed+Central%22">BioMed Central </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101610673 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2051-5960 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2220515960%22">20515960 </searchLink><i>NLM ISO Abbreviation: </i>Acta Neuropathol Commun <i>Subsets: </i>MEDLINE
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