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Academic Journal

Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications.

  • Authors : Wu YF; Institute of Molecular Biology, Academia Sinica, Taipei, Taiwan. .; Neuroscience Program of Academia Sinica, Academia Sinica, Taipei, Taiwan. .

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/trends ; Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/therapy

  • Source: Journal of biomedical science [J Biomed Sci] 2025 Feb 21; Vol. 32 (1), pp. 30. Date of Electronic Publication: 2025 Feb 21.Publisher: BioMed Central Country of Publication: England NLM ID: 9421567 Publication Model: Electronic Cited Medium: Internet ISSN: 1423-0127

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Academic Journal

Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy.

  • Authors : Zwi-Dantsis L; Department of Mechanical Engineering, Roberts Building, University College London, London WC1E 6BT, UK.; Mohamed S

Subjects: Dependovirus*/Dependovirus*/Dependovirus*/genetics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Genetic Vectors*

  • Source: Viruses [Viruses] 2025 Feb 09; Vol. 17 (2). Date of Electronic Publication: 2025 Feb 09.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101509722 Publication Model: Electronic Cited Medium: Internet ISSN: 1999-4915

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Academic Journal

Endothelial and neuronal engagement by AAV-BR1 gene therapy alleviates neurological symptoms and lipid deposition in a mouse model of Niemann-Pick type C2.

  • Authors : Rasmussen CLM; Neurobiology Research and Drug Delivery, Department of Health Science and Technology, Aalborg University, Selma Lagerlöfts Vej 249, 9260, Gistrup, Denmark.; Frederiksen SF

Subjects: Dependovirus*/Dependovirus*/Dependovirus*/genetics ; Niemann-Pick Disease, Type C*/Niemann-Pick Disease, Type C*/Niemann-Pick Disease, Type C*/therapy ; Niemann-Pick Disease, Type C*/Niemann-Pick Disease, Type C*/Niemann-Pick Disease, Type C*/metabolism Niemann-Pick disease, type C2

  • Source: Fluids and barriers of the CNS [Fluids Barriers CNS] 2025 Jan 31; Vol. 22 (1), pp. 13. Date of Electronic Publication: 2025 Jan 31.Publisher: Biomed Central Country of Publication: England NLM ID: 101553157 Publication Model: Electronic Cited Medium: Internet ISSN: 2045-8118

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Academic Journal

Advances in Gene Therapy for Rare Diseases: Targeting Functional Haploinsufficiency Through AAV and mRNA Approaches.

  • Authors : Bara-Ledesma N; Internal Medicine Department, Hospital Universitario Ramón y Cajal, IRYCIS, 28034 Madrid, Spain.; Faculty of Medicine and Health Sciences, Universidad de Alcalá (UAH), 28805 Alcalá de Henares, Spain.

Subjects: Dependovirus*/Dependovirus*/Dependovirus*/genetics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Haploinsufficiency*/Haploinsufficiency*/Haploinsufficiency*/genetics

  • Source: International journal of molecular sciences [Int J Mol Sci] 2025 Jan 11; Vol. 26 (2). Date of Electronic Publication: 2025 Jan 11.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Academic Journal

AAV-mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives.

  • Authors : Zhang L; State Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.; Tan F

Subjects: Deafness*/Deafness*/Deafness*/genetics ; Deafness*/Deafness*/Deafness*/therapy ; Dependovirus*/Dependovirus*/Dependovirus*/genetics

  • Source: Advanced science (Weinheim, Baden-Wurttemberg, Germany) [Adv Sci (Weinh)] 2024 Dec; Vol. 11 (47), pp. e2402166. Date of Electronic Publication: 2024 Nov 18.Publisher: WILEY-VCH Country of Publication: Germany NLM ID: 101664569 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2198-3844

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Academic Journal

In Vivo Selection of S/MAR Sequences to Favour AAV Episomal Maintenance in Dividing Cells.

  • Authors : Llanos-Ardaiz A; Vivet Therapeutics S.L., 31008 Pamplona, Spain.; DNA & RNA Medicine Division, Centre for Applied Medical Research (CIMA), University of Navarra, 31009 Pamplona, Spain.

Subjects: Dependovirus*/Dependovirus*/Dependovirus*/genetics ; Genetic Vectors*/Genetic Vectors*/Genetic Vectors*/genetics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods

  • Source: International journal of molecular sciences [Int J Mol Sci] 2024 Nov 27; Vol. 25 (23). Date of Electronic Publication: 2024 Nov 27.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Academic Journal

Adeno-Associated Viral Vectors in the Treatment of Epilepsy.

  • Authors : Mullagulova AI; Institute for Fundamental Medicine and Biology, Kazan Federal University, Kazan 420008, Russia.; Timechko EE

Subjects: Dependovirus*/Dependovirus*/Dependovirus*/genetics ; Epilepsy*/Epilepsy*/Epilepsy*/therapy ; Epilepsy*/Epilepsy*/Epilepsy*/genetics

  • Source: International journal of molecular sciences [Int J Mol Sci] 2024 Nov 11; Vol. 25 (22). Date of Electronic Publication: 2024 Nov 11.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Academic Journal

Prevalence of Antibodies against Adeno-Associated Viruses (AAVs) in Göttingen Minipigs and Its Implications for Gene Therapy and Xenotransplantation.

  • Authors : Jacobsen KR; Ellegaard Göttingen Minipigs A/S, 4261 Dalmose, Denmark.; Mota J

Subjects: Dependovirus*/Dependovirus*/Dependovirus*/immunology ; Dependovirus*/Dependovirus*/Dependovirus*/genetics ; Swine, Miniature*

  • Source: Viruses [Viruses] 2024 Oct 15; Vol. 16 (10). Date of Electronic Publication: 2024 Oct 15.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101509722 Publication Model: Electronic Cited Medium: Internet ISSN: 1999-4915

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Academic Journal

A Comparative Analysis of Models for AAV-Mediated Gene Therapy for Inherited Retinal Diseases.

  • Authors : Alsalloum A; Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, Russia.; Gornostal E

Subjects: Dependovirus*/Dependovirus*/Dependovirus*/genetics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Retinal Diseases*/Retinal Diseases*/Retinal Diseases*/genetics

  • Source: Cells [Cells] 2024 Oct 15; Vol. 13 (20). Date of Electronic Publication: 2024 Oct 15.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409

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Academic Journal

A comprehensive review of AAV-mediated strategies targeting microglia for therapeutic intervention of neurodegenerative diseases.

  • Authors : Zhou L; Henan Neurodevelopment Engineering Research Center for Children, Children's Hospital Affiliated to Zhengzhou University, Henan Children's Hospital Zhengzhou Children's Hospital, Zhengzhou, 450018, China. .; Wang Y

Subjects: Microglia*/Microglia*/Microglia*/metabolism ; Neurodegenerative Diseases*/Neurodegenerative Diseases*/Neurodegenerative Diseases*/therapy ; Dependovirus*/Dependovirus*/Dependovirus*/genetics

  • Source: Journal of neuroinflammation [J Neuroinflammation] 2024 Sep 19; Vol. 21 (1), pp. 232. Date of Electronic Publication: 2024 Sep 19.Publisher: BioMed Central Country of Publication: England NLM ID: 101222974 Publication Model: Electronic Cited Medium: Internet ISSN: 1742-2094

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  • 1-10 of  3,310 results for ""Genetic Therapy""