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4 articles for “Spinal muscular atrophy”
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Computational Investigation of Withania somnifera Compounds Targeting S. pombe SMN YG-Dimer in Spinal Muscular Atrophy: Insights from Molecular Docking Analysis
Abstract: Objectives: The primary objective of this study is to determine the potential of phytochemical constituents of Withania somnifera in treating spinal muscular atrophy, an autosomal recessive genetic neurodegenerative disorder, through computational techniques. To investigate the binding affinity of phytocompound to the target protein S. pombe SMN YG-Dimer through molecular docking. Besides, evaluating the pharmacological attributes of phytochemical constituents to assess their ability as an ideal prospect for developing therapeutic medication. …
Published in Research and Reviews : Journal of Computational Biology · Vol. 13, Issue 2, 2024 · pp. 39–52 Read article
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Gene Therapy in Modern Medicine: Promises and Challenge in Treating Genetic Diseases
Abstract: Gene therapy is a medical approach that focuses on altering or adjusting an individual’s genes to treat or prevent illnesses. The aim is to repair faulty genes or insert new ones into the body to combat diseases. Gene therapy can involve directly introducing modified or new genes into a patient’s cells or altering the genes already present in the patient’s body. This approach shows potential for treating a range of …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 2, Issue 2, 2024 · pp. 27–32 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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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