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3 articles for “Prime 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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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
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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