Gene therapy
12 articles · search the full text for this term
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RNA- Based Drug Delivery, Current Advances, Pharmaceutical Applications, and Future Perspectives
Abstract: RNA-based therapeutics have emerged as a transformative approach in modern medicine due to their ability to regulate gene expression with high specificity and precision. Advances in molecular biology, RNA chemistry, and nanotechnology have accelerated the development of diverse RNA modalities, including messenger RNA (mRNA), small interfering RNA (siRNA), microRNA (miRNA), antisense oligonucleotides (ASOs), and RNA aptamers. These therapeutics offer promising strategies for the treatment of cancer, genetic disorders, infectious diseases, …
Published in Research & Reviews: A Journal of Drug Design & Discovery · Vol. 13, Issue 2, 2026 Read article
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Genetic Counseling: A Pathway to Understanding and Managing Inherited Health Risks
Abstract: Genetic counseling is an essential aspect of personalized medicine that supports individuals and families in understanding the implications of inherited health conditions. With the rising prevalence of genetic disorders and advancements in genomic technologies, counseling provides critical insights into risk factors, diagnostic options, and informed decision-making. This paper explores the classification of genetic disorders, major genetic and environmental causes, and the expanding role of genetic counseling within India’s culturally diverse …
Published in International Journal of Evidence Based Nursing And Practices · Vol. 4, Issue 1, 2026 · pp. 10–14 Read article
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Biological Membranes: Structure, Function, and Their Roles in Disease Pathophysiology
Abstract: Biological membranes are important structures that control cellular work, signal transmission, transport, and communication. Dysfunction of membranes has been considered in the development of many diseases, such as cancer, neurodegenerative diseases, and infections, with the distortion of lipid composition, protein activity, and cellular connections involved in the pathogenesis of the disease. The damage on membrane can be caused by oxidative stress, genetic mutation, and environmental influence to disrupt cellular homeostasis. …
Published in International Journal of Membranes · Vol. 3, Issue 1, 2026 · pp. 38–46 Read article
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CRISPR-Cas9: Revolutionizing the Genetic Frontier
Abstract: The rapid development of CRISPR/CRISPR-associated enzyme (Cas) technology has enabled truly customised treatment of human genetic disorders, paving the way for recent developments in the field of gene therapy. Because CRISPR/Cas can accurately target and edit individual genes within a genome, it has established itself as a formidable tool for genetic manipulation. CRISPR/Cas9 technology allows for precise editing of specific DNA sequences in an organism's genome. This method uses three …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 3, Issue 2, 2025 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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Emerging Therapies in Respiratory Tract Infections: Current Advances and Future Perspectives
Abstract: The impact of respiratory tract infections (RTI) as a global public health problem continues to be high morbidity and mortality rates. The development of novel therapeutic approaches stemmed from the fact that conventional treatment strategies, antibiotics, antivirals, antifungals etc. have become increasingly ineffective because of rise of antimicrobial resistance (AMR). Next generation therapies, such as novel antimicrobial agents, host directed therapies and novel drug delivery systems have come on the …
Published in Recent Trends in Infectious Diseases · Vol. 2, Issue 2, 2025 · pp. 17–25 Read article
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An Overview of Gene Therapy
Abstract: Gene therapy is a revolutionary technique in medical science that seeks to cure or stop illnesses by means of introducing, altering, or quieting genes inside a patient's cells. Advancements in molecular biology, genetics, and biotechnology have propelled this field forward markedly changed over the last few decades. The underlying idea of gene therapy is to introduce genetic material into target cells via viral or non-viral vectors to rectify faulty genes, …
Published in Research and Reviews: A Journal of Pharmaceutical Science · Vol. 16, Issue 3, 2025 · pp. 89–105 Read article
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A review on Design of Gene Therapy and Its Potential for Craniofacial Regeneration
Abstract: One of the most talked-about topics of the 21st century is gene therapy, which holds the promise of treating many diseases. Current gene therapy research explores a wide range of potential treatments, such as enhancing the body’s immune response to tumors, promoting the formation of new blood vessels in the heart to mitigate heart attacks, and preventing HIV replication in AIDS patients. Gene therapy involves the introduction, alteration, or replacement …
Published in Emerging Trends in Personalized Medicines · Vol. 2, Issue 2, 2025 · pp. 6–12 Read article
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Exhaustive Search Meets DNA Sequencing: A Comprehensive Review of TSP-Based Approaches
Abstract: Exhaustive search is a highly computational complex algorithm that checks every possibility to obtain the best solution. We illustrate an exhaustive search by applying it to three important problems: the traveling salesman problem, the knapsack problem, and the assignment problem. In this paper, we took a traveling salesman problem to explain DNA Sequencing. Since traveling salesman problem is an algorithmic problem that finds the shortest route between a set of …
Published in International Journal of Bioinformatics and Computational Biology · Vol. 2, Issue 2, 2024 · pp. 11–21 Read article
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Wiskott-Aldrich Syndrome: A Comprehensive Guide
Abstract: Wiskott-Aldrich Syndrome (WAS) is an uncommon genetic disorder inherited through the X chromosome, marked by a combination of immune system deficiencies, eczema, and low platelet counts. This syndrome primarily affects males, leading to significant morbidity and mortality due to recurrent infections, bleeding complications, and autoimmune diseases. Mutations in the WAS gene disrupt the production of the Wiskott-Aldrich Syndrome protein (WASp), crucial for the functioning of immune cells and platelet formation. …
Published in Research and Reviews : A Journal of Immunology · Vol. 14, Issue 2, 2024 · pp. 39–46 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