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6 articles for “CRISPR-Cas9 gene editing”
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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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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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Gene Editing in Dairy Cows: Advancing Milk Yield, Health, and Resilience for Sustainable Dairy Development
Abstract: Gene editing has emerged as a transformative tool in dairy science, offering unprecedented opportunities to enhance productivity, improve animal health, and bolster resilience against environmental challenges. This study explores the potential of gene editing technologies, including CRISPR-Cas9, in advancing milk yield, optimizing milk composition, and addressing genetic disorders in dairy cows. Specific applications include targeting genes associated with mastitis resistance, improving feed efficiency, and introducing heat tolerance traits to adapt …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 3, Issue 2, 2025 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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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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Revolutionizing Blood Cancer Treatment: Emerging Therapies and Future Perspectives
Abstract: Blood cancer inclusive of leukemia, lymphoma and multiple myeloma poses a significant threat in the world today because of its multifactorial process and its non-amenable to standard therapies. Some of such approaches are immunotherapy, gene-editing technology and targeted small molecule inhibitors that have altered the nature of treatments. CAR T cell therapy is a molecular targeted therapy for cancers and several clinical studies have reported a huge success. Also, the …
Published in International Journal of Biomedical Innovations and Engineering · Vol. 3, Issue 2, 2025 Read article