Search
11 articles for “CRISPR-Cas9 gene editing”
-
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
-
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
-
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
-
Innovations in Gene Therapy: Revolutionizing Treatment for Genetic Disorders and Diseases
Abstract: Gene therapy is a revolutionary intervention in the treatment of genetic diseases since it is an intervention that aims at the very source of diseases, through editing and modification of genes. More recent innovations in gene editing systems, such as CRISPR-Cas9, CRISPR-Cas12, and prime editing, have greatly increased the accuracy and effectiveness of gene therapies, enabling more specific therapies. The opportunities of personalized medicine are also extended by the creation …
Published in Research and Reviews : A Journal of Biotechnology · Vol. 16, Issue 1, 2026 Read article
-
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
-
Gene Edited Livestock for Enhanced Feed Efficiency and Metabolic Adaptation Through Precision Genomic Interventions
Abstract: Advances in gene editing technologies have transformed the landscape of livestock production by enabling precise manipulation of genetic material associated with productivity, health, and environmental adaptability. Tools such as CRISPR Cas9 and emerging next generation editors allow targeted modifications that improve feed efficiency, growth performance, nutrient utilization, and stress tolerance in farm animals. This review highlights the genetic basis of feed efficiency and metabolic regulation, focusing on key genes and …
Published in Research and Reviews : A Journal of Biotechnology · Vol. 16, Issue 2, 2026 Read article
-
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
-
Role of Crispr-Cas9 in Plants
Abstract: The innovative CRISPR Cas9 system has emerged as a game changing genetic engineering tool in years offering levels of precision, efficiency and flexibility in editing the genomes of various organisms, including plants. This summary aims to provide an examination of the role of CRISPR Cas9, in modifying plant genomes highlighting its benefits, drawbacks and possible uses. Thanks to its versatility and user-friendly nature CRISPR Cas9 technology has expedited studies in …
Published in Research and Reviews : A Journal of Biotechnology · Vol. 14, Issue 3, 2024 · pp. 1–10 Read article
-
A Brief Review On Nanoparticles Drug Delivery System Used In Cervical Cancer
Abstract: Among cervical tumor-related deaths worldwide, cervical cancer is a major cause. The limitations of traditional methods, such as chemotherapy and radiation therapy, stem from their adverse effects and increased susceptibility to medications. Despite being seen as innovative options, immune checkpoint inhibitors (ICIs) have rather low clinical response rates. Reliable treatments for patients with metastatic or recurring cervical cancer are currently lacking. Lately, nanomaterials including polymers, liposomes, and dendrimers have been …
Published in Research and Reviews : A Journal of Life Sciences · Vol. 15, Issue 1, 2025 · pp. 30–40 Read article
-
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
-
Role of Epigenetic Mechanisms in Lung Fibrosis: Therapeutic Opportunities and Challenges
Abstract: Lung fibrosis poses a serious risk to one's health since it might result in respiratory failure due to an abnormal accumulation of fibrotic tissue in the lungs. Epigenetic mechanisms, such as histone changes, DNA methylation, and non-coding RNAs, closely control gene expression patterns and cellular processes associated with lung fibrosis. Anomalies in DNA methylation patterns connected to changes in gene expression profiles and disturbances in fibrotic signalling pathways are potential …
Published in Research and Reviews : A Journal of Medical Science and Technology · Vol. 13, Issue 2, 2024 · pp. 9–20 Read article