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5 articles for “Monogenic diseases”
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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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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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Genomic Medicine Revolutionizes Heart Care: A New Standard of Practice
Abstract: The integration of genomic and precision medicine into cardiovascular healthcare represents a transformative advancement in addressing the global burden of cardiovascular diseases (CVDs). Precision medicine leverages genomic, proteomic, and metabolomic data to provide personalized care, optimizing treatment outcomes while minimizing adverse effects. Over the past decade, extensive research has highlighted its potential to revolutionize the management of major CVDs, including myocardial infarction, hypertension, and heart failure, which significantly contribute to …
Published in International Journal of Tropical Medicines · Vol. 2, Issue 2, 2025 · pp. 30–37 Read article
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Prime Editing and Base Editing in Human Hematopoietic Stem Cells Toward Scarless Correction of Monogenic Blood Disorders
Abstract: Monogenic hematological disorders – such as sickle cell disease (SCD), β-thalassemia, and X-linked chronic granulomatous disease (X-CGD) – affect >400, 000 newborns worldwide each year, with a considerable burden in low-resource settings . Although donor-derived allogeneic hematopoietic stem-cell transplantation (HSCT) is widely regarded as curative, its application is limited due to issues of donor availability and graft-versus-host disease (GVHD) and conditioning-related toxicity. DSB-based conventional CRISPR-Cas9 strategies are limited by inefficient …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 4, Issue 1, 2026 Read article
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Crispr Cas – Revolutionizing Modern Therapies and Beyond
Abstract: CRISPR-Cas technology has emerged as a transformative tool in modern molecular biology, revolutionizing both fundamental research and clinical applications. This RNA-guided gene-editing system enables precise and efficient genomic modifications, offering unprecedented potential for addressing genetic disorders, infectious diseases, and oncological conditions through innovative therapeutic interventions. The inherent specificity and programmability of CRISPR-Cas systems have facilitated breakthroughs in diverse fields, including precision medicine, regenerative therapies, and immuno-oncology. Beyond its therapeutic applications, …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 3, Issue 1, 2025 · pp. 25–38 Read article