Toward a gene therapy for neurological and somatic MPSIIIA

Virginia Haurigot Center of Animal Biotechnology and Gene Therapy (CBATEG) and Department of Biochemistry and Molecular Biology; School of Veterinary Medicine; Universitat Autònoma de Barcelona; Barcelona, Spain

Fatima Bosch Center of Animal Biotechnology and Gene Therapy (CBATEG) and Department of Biochemistry and Molecular Biology; School of Veterinary Medicine; Universitat Autònoma de Barcelona; Barcelona, SpainCorrespondence[email protected]

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Tierra A. Bobo, Preston N. Samowitz, Michael I. Robinson & Haiyan Fu. (2020) Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector. Molecular Therapy - Methods & Clinical Development 19, pages 474-485.
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Joseph Edward Rittiner, Malik Moncalvo, Ornit Chiba-Falek & Boris Kantor. (2020) Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases. Frontiers in Molecular Neuroscience 13.
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Haiyan Fu, Kim Zaraspe, Naoko Murakami, Aaron S. Meadows, Ricardo J. Pineda, Douglas M. McCarty & Joseph Muenzer. (2018) Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice. Molecular Therapy - Methods & Clinical Development 10, pages 327-340.
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Haiyan FuAaron S. Meadows, Ricardo J. Pineda, Krista L. Kunkler, Kristen V. TruxalKim L. McBrideKevin M. FlaniganDouglas M. McCarty. (2017) Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. Human Gene Therapy Clinical Development 28:4, pages 187-196.
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Haiyan FuAaron S MeadowsRicardo J PinedaKrista L KunklerKristen V TruxalKim L McBrideKevin FlaniganDouglas M McCarty. (2017) Differential prevalence of antibodies against adeno-associated virus in healthy children and patients with mucopolysaccharidosis III: perspective for AAV-mediated gene therapy. Human Gene Therapy Clinical Development.
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Carles Roca, Sandra Motas, Sara Marcó, Albert Ribera, Víctor Sánchez, Xavier Sánchez, Joan Bertolin, Xavier León, Jennifer Pérez, Miguel Garcia, Pilar Villacampa, Jesús Ruberte, Anna Pujol, Virginia Haurigot & Fatima Bosch. (2017) Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID. Human Molecular Genetics 26:8, pages 1535-1551.
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Elena L. Aronovich & Perry B. Hackett. (2015) Lysosomal storage disease: Gene therapy on both sides of the blood–brain barrier. Molecular Genetics and Metabolism 114:2, pages 83-93.
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Toward a gene therapy for neurological and somatic MPSIIIA

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Toward a gene therapy for neurological and somatic MPSIIIA

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