Dual Purpose Vectors for Rare Neurological Diseases: Molecular Therapy
Descrição
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The intersection of vector biology, gene therapy, and hemophilia - Research and Practice in Thrombosis and Haemostasis
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Precision medicine in rare diseases: What is next? - Tesi - 2023 - Journal of Internal Medicine - Wiley Online Library
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Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
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Dual role of the nasal microbiota in neurological diseases—An unignorable risk factor or a potential therapy carrier - ScienceDirect
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Frontiers Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord
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Full article: Gene therapy for neurological disorders: challenges and recent advancements
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Network analysis reveals rare disease signatures across multiple levels of biological organization
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Gene specific therapies – the next therapeutic milestone in neurology, Neurological Research and Practice
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Coave Therapeutics – Coave Therapeutics is a clinical stage biotechnology company focused on developing life changing gene therapies in rare ocular and CNS (Central Nervous System) diseases.
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Current Clinical Applications of In Vivo Gene Therapy with AAVs: Molecular Therapy
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Antioxidants, Free Full-Text
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Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease: Molecular Therapy - Methods & Clinical Development
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Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice: Molecular Therapy
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Gene therapy - Wikipedia
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Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain
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