Refining strategies to translate genome editing to the clinic
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Zinc finger nuclease
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Zinc finger nuclease
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Genome editing has been well established as a genome engineering tool that enables researchers to establish causal linkages between genetic mutation and biological phenotypes, providing further understanding of the genetic manifestation of many debilitating diseases. More recently, the paradigm of genome editing technologies has evolved to include the correction of mutations that cause diseases via the use of nucleases such as zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALENs), and more recently, Cas9 nuclease. With the aim of reversing disease phenotypes, which arise from somatic gene mutations, current research focuses on the clinical translatability of correcting human genetic diseases in vivo, to provide long-term therapeutic benefits and potentially circumvent the limitations of in vivo cell replacement therapy. In this review, in addition to providing an overview of the various genome editing techniques available, we have also summarized several in vivo genome engineering strategies that have successfully demonstrated disease correction via in vivo genome editing. The various benefits and challenges faced in applying in vivo genome editing in humans will also be discussed.
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Manipulating genomes by traditional targeted genome editing technique( gene targeting) is inefficient,making it impractical or difficult to use the technique as a gene-therapy approach to cure diseases and decipher gene functions. To overcome this problem,next-generation targeted gene-editing techniques were developed to achieve higher efficiency for gene correction,specific locus integration or knock-in and high throughput gene knock-out. The progress of new techniques for targeted genome-editing tools were reviewed, including zinc finger nucleases( ZFN), transcription activator-like effector nucleases( TALENs), and a clustered regularly interspaced short palindromic repeats( CRISPR) / Cas system. A brief summary of the history, recent structure,progress,and future prospects was presented. After comparing these tools,it was found that CRISPR systems offer an advantage over ZFN and TALEN.
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Genome editing with engineered nucleases is an emerging technology that enables manipulation of targeted genes in many organisms and cell lines. To date, two types of engineered nucleases have been developed. Zinc finger nucleases (ZFNs), which first emerged in 1996, have a long and successful history of genome editing. Transcription activator-like effector nucleases (TALENs), on the other hand, have been recently developed as a more user-friendly engineered nuclease, because TALENs are easy to construct in-house and the target site may be selected in any gene. Using these engineered nucleases, targeted gene disruptions have been achieved successfully in cultured cells and various organisms. Furthermore, gene correction or targeted gene addition have been reported mainly in cultured cells, and also in several organisms. Surprisingly, a third genome editing technology, known as CRISPR/Cas system, suddenly emerged in 2013 and efficient genome editing using this system was reported in various organisms. In this way, genome editing technology is now becoming the most exciting field in life sciences and important for developmental biology. We thought that it is a good timing to publish a special issue focusing on targeted genome editing. We hope this issue will provide general information on available techniques and materials for developmental biologists.
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The ongoing advent of genome editing with programmable nucleases, including zinc-finger nuclease (ZFN), TAL effector nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-associated RNA-guided endonuclease Cas9 (CRISPR/Cas9), have spurred the hematopoietic stem cell gene therapy (HSC-GT). In particular, CRISPR/Cas9-mediated gene editing revealed promising outcomes in several preclinical disease models including inherited and neoplastic hematological diseases. In this review, we focused on the utilization of the CRISPR/Cas9 system as a possible treatment option for hemoglobinopathies and hematological tumors. We summarize the recent advances with CRISPR/Cas9 and its therapeutic potential for genome editing in cells from hematopoietic origin. We also critically discussed the limitations inherent to the CRISPR/Cas9 and possible alternatives for the improvement of genome editing.
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The development of custom-designed nucleases (CDNs), including zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), has made it possible to perform precise genetic engineering in many cell types and species. More recently, clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has been successfully employed for genome editing. These RNA-guided DNA endonucleases are shown to be more efficient and flexible than CDNs and hold great potential for applications in both biological studies and medicine. Here, we review the progress that has been made for all three genome editing technologies in modifying both cells and model organisms, compare important aspects of each approach, and summarize the applications of these tools in disease modeling and gene therapy. In the end, we discuss future prospects of the field.
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The most significant challenges to improving yield in agriculture science need progressive improvement with novel strategies. The modern gene editing scenario must focus on reducing the infectious plant diseases caused by plant pathogens. Targeted plant genome engineering enables optimal modifications in commercial plant varieties by incorporating effective alternative gene editing methods over other traditional approaches. Genome editing technology is a multipurpose approach that rapidly progressed in plant biology to alter the phenotype and genotype of the species. Despite its advantages, traditional genome modification could not meet the recent demands for site-directed mutagenesis, high effectiveness, and retrofitting of artificial endonucleases 3 . Current gene editing technologies have been subdivided into three kinds of cleavage systems, which are Transcription activator-like effector nucleases TALEN, Zinc Finger nucleases ZFN, CRISPR Cas (Clustered Regularly Interspaced Short Palindromic Repeats - CRISPR associated protein). These methods lead to single-stranded or double-stranded mutation through homologous recombination. Hence, this Review is motivated by the advantages of the gene editing tools in developing the plant immune system.
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