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4 articles for “gene therapy and adeno-associated virus”
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Hemophilia and Gene Therapy–Review
Abstract: The coagulation factor VIII or factor IX genes are affected by the monogenic mutational illness known as hemophilia. Recombinant clotting factors, which are secure and efficient, are the preferred palliative therapy. In order to ensure steady and long-lasting concentrations of the damaged circulating factor, advanced therapies will be curative. Adeno-associated vectors have primarily been used, and the results have been positive in terms of amounts and timings of expression. These …
Published in Research and Reviews : A Journal of Medical Science and Technology · Vol. 12, Issue 3, 2024 · pp. 1–5 Read article
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Gene Therapy 2.0: Harnessing Cutting-Edge Technologies for Precision Medicine
Abstract: Somatic gene therapy has become the standard form of gene therapy; this creates the Gene Therapy 2.0 era. This new and distinct time consists in the new-generation genome-editing tools and markers, more efficient systems for gene delivery, and personalized medicine approaches that raise the efficacy and safety of the treatment. Base and prime editing are two types of CRISPR-based gene editing tools have provided high precision and reduced off-target effects …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 3, Issue 2, 2025 Read article
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Advancing Gene Therapy: Next-Generation Viral Vector Engineering for Precision, Safety, and Scalability
Abstract: Gene therapy has emerged as a paradigm-shifting modality for the treatment of genetic disorders, malignancies, and rare diseases through the delivery of therapeutic nucleic acids aimed at correcting or modulating dysfunctional gene expression. Among the various delivery systems, viral vectors including adeno-associated viruses (AAVs), lentiviruses, adenoviruses, retroviruses, and herpes simplex viruses have proven indispensable owing to their high transduction efficiencies and adaptability. This review offers a comprehensive assessment of viral …
Published in International Journal of Virus Studies · Vol. 2, Issue 2, 2025 · pp. 37–55 Read article
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An AAV Vector for Inducible Gene Expression Preferentially in Muscles
Abstract: Adeno associated viral (AAV) vectors has been used widely in gene therapy and efforts have been made to improve their utility by adding genetic elements that would enable targeting transgene expression to particular cells or tissues of interest and permitting on/off regulation of expression. In this study, we designed a recombinant AAV9 variant PHP.eB vector with muscle creatine kinase (Mck)-derived enhancers, a synthetic muscle-expression promoter, in combination with a third-generation …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 1, Issue 2, 2023 · pp. 46–61 Read article