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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

Gene Therapy: An Historical Overview for Familial Hearing Loss.

  • Authors : Guthrie OW; Cell & Molecular Pathology Laboratory, Department of Communication Sciences and Disorders, Northern Arizona University, Flagstaff, AZ 86011, USA.

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/history ; Hearing Loss*/Hearing Loss*/Hearing Loss*/therapy

  • Source: International journal of molecular sciences [Int J Mol Sci] 2025 Feb 10; Vol. 26 (4). Date of Electronic Publication: 2025 Feb 10.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Academic Journal

The role of hospital pharmacists in supporting the appropriate and safe use of CGT/ATMPs: a scoping review of current insights.

  • Authors : Shi J; State Key Laboratory of Quality Research in Chinese Medicine, Institute of Chinese Medical Sciences, University of Macau, Macao SAR, China.; Chen X

Subjects: Cell- and Tissue-Based Therapy*/Cell- and Tissue-Based Therapy*/Cell- and Tissue-Based Therapy*/adverse effects ; Cell- and Tissue-Based Therapy*/Cell- and Tissue-Based Therapy*/Cell- and Tissue-Based Therapy*/methods ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/adverse effects

  • Source: BMC health services research [BMC Health Serv Res] 2025 Jan 09; Vol. 25 (1), pp. 52. Date of Electronic Publication: 2025 Jan 09.Publisher: BioMed Central Country of Publication: England NLM ID: 101088677 Publication Model: Electronic Cited Medium: Internet ISSN: 1472-6963

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Academic Journal

Emerging Gene Therapy Based on Nanocarriers: A Promising Therapeutic Alternative for Cardiovascular Diseases and a Novel Strategy in Valvular Heart Disease.

  • Authors : Yang H; Laboratory of Cardiac Structure and Function, Institute of Cardiovascular Diseases, West China Hospital, Sichuan University, Chengdu 610041, China.; Department of Cardiology, West China Hospital, Sichuan University, No.37 Guoxue Street, Chengdu 610041, China.

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Heart Valve Diseases*/Heart Valve Diseases*/Heart Valve Diseases*/therapy ; Heart Valve Diseases*/Heart Valve Diseases*/Heart Valve Diseases*/genetics

  • Source: International journal of molecular sciences [Int J Mol Sci] 2025 Feb 18; Vol. 26 (4). Date of Electronic Publication: 2025 Feb 18.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Academic Journal

From Origin to the Present: Establishment, Mechanism, Evolutions and Biomedical Applications of the CRISPR/Cas-Based Macromolecular System in Brief.

  • Authors : Yuan Z; Institute of Chinese Materia Medica, China Academy of Chinese Medical Sciences, Beijing 100022, China.

Subjects: CRISPR-Cas Systems* ; Gene Editing*/Gene Editing*/Gene Editing*/methods ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods

  • Source: Molecules (Basel, Switzerland) [Molecules] 2025 Feb 18; Vol. 30 (4). Date of Electronic Publication: 2025 Feb 18.Publisher: MDPI Country of Publication: Switzerland NLM ID: 100964009 Publication Model: Electronic Cited Medium: Internet ISSN: 1420-3049

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Academic Journal

Analysis of Hepatic Lentiviral Vector Transduction: Implications for Preclinical Studies and Clinical Gene Therapy Protocols.

  • Authors : Hu P; Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.; Hao Y

Subjects: Genetic Vectors*/Genetic Vectors*/Genetic Vectors*/genetics ; Transduction, Genetic* ; Lentivirus*/Lentivirus*/Lentivirus*/genetics

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

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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

Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones.

  • Authors : Hwu WL; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, National Taiwan University College of Medicine, Taipei, Taiwan.; Lee HM

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods; Humans ; Female Aromatic amino acid decarboxylase deficiency

  • Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2025 Feb 07; Vol. 20 (1), pp. 58. Date of Electronic Publication: 2025 Feb 07.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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Academic Journal

Regulatory Elements for Gene Therapy of Epilepsy.

  • Authors : Chesnokova E; Laboratory of Cellular Neurobiology of Learning, Institute of Higher Nervous Activity and Neurophysiology of the Russian Academy of Sciences, Moscow 117485, Russia.; Bal N

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Epilepsy*/Epilepsy*/Epilepsy*/genetics ; Epilepsy*/Epilepsy*/Epilepsy*/therapy

  • Source: Cells [Cells] 2025 Feb 06; Vol. 14 (3). Date of Electronic Publication: 2025 Feb 06.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409

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Academic Journal

30 Years Since the Proposal of Exon Skipping Therapy for Duchenne Muscular Dystrophy and the Future of Pseudoexon Skipping.

  • Authors : Matsuo M; Graduate School of Science, Technology and Innovation, Kobe University, Kobe 657-8501, Japan.

Subjects: Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/therapy ; Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/genetics ; Exons*

  • Source: International journal of molecular sciences [Int J Mol Sci] 2025 Feb 03; Vol. 26 (3). Date of Electronic Publication: 2025 Feb 03.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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  • 1-10 of  34,671 results for ""Genetic Therapy""