The genome editing toolbox: a spectrum of approaches for targeted modification

Cheng, J.; Alper, Hal S.

doi:10.1016/j.copbio.2014.06.005

Cited by 32 publications

(25 citation statements)

References 53 publications

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“…Future breakthroughs in the field are likely to come from analysis of renal function in models of channelopaties with animals in which precise modification of genes encoding proteins, involved in ET-1-mediated ENaC inhibition has been achieved. The recent advances in targeted genome editing techniques suggest that this task could be achieved within next few years (Cheng & Alper, 2014). …”

Section: Discussionmentioning

confidence: 99%

Inhibition of ENaC by Endothelin-1

Sorokin

Staruschenko

2015

Hormones and Transport Systems

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The amiloride-sensitive epithelial Na+ channel (ENaC) is a key player in the regulation of Na+ homeostasis. Its functional activity is under continuous control by a variety of signaling molecules including bioactive peptides of endothelin family. Since ENaC dysfunction is causative for disturbances in total body Na+ levels associated with abnormal regulation of blood volume, blood pressure, and lung fluid balance, the uncovering the molecular mechanisms of inhibitory modulation or inappropriate activation of ENaC is crucial for the successful treatment of a variety of human diseases including hypertension. The precise regulation of ENaC is particularly important for normal Na+ and fluid homeostasis in organs where endothelins are known to act: kidneys, lung and colon. Inhibition of ENaC by endothelin-1 (ET-1) has been established in renal cells and several molecular mechanisms of inhibition of ENaC by ET-1 are proposed and will be reviewed in this chapter.

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Section: Discussionmentioning

confidence: 99%

Inhibition of ENaC by Endothelin-1

Sorokin

Staruschenko

2015

Hormones and Transport Systems

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“…The second group, transcription activator–like effector nucleases (TALENs), were popular, as they dramatically increased the availability of target sites, allowing almost any sequence to be targeted. However, the construction of TALENs is challenging and time consuming . Both ZFNs and TALENs use DNA‐binding proteins to tether endonuclease catalytic domains to specific genomic loci.…”

Section: Introductionmentioning

confidence: 99%

Understanding α‐globin gene regulation and implications for the treatment of β‐thalassemia

Mettananda

Gibbons

Higgs

2015

Annals of the New York Academy of Sciences

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Over the past three decades, a vast amount of new information has been uncovered describing how the globin genes are regulated. This knowledge has provided significant insights into the general understanding of the regulation of human genes. It is now known that molecular defects within and around the α- and β-globin genes, as well as in the distant regulatory elements, can cause thalassemia. Unbalanced production of globin chains owing to defective synthesis of one, and the continued unopposed synthesis of another, is the central causative factor in the cellular pathology and pathophysiology of thalassemia. A large body of clinical, genetic, and experimental evidence suggests that altering globin chain imbalance by reducing the production of α-globin synthesis ameliorates the disease severity in patients with β-thalassemia. With the development of new genetic-based therapeutic tools that have a potential to decrease the expression of a selected gene in a tissue-specific manner, the possibility of decreasing expression of the α-globin gene to improve the clinical severity of β-thalassemia could become a reality.

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“…Concurrent multiplex mutagenesis was shown in rice protoplasts and transgenic plants for various quantities of sgRNAs (up to eight) in a tRNA sgRNA exhibit. Researchers from KAUST in Saudi Arabia showed the capability of plant infections for multiplex genome building [7][8][9]. By utilizing the Tobacco shake infection (TRV) to convey sgRNAs to transgenic N. benthamiana that steadily over-communicated Cas9.…”

Section: Crispr-cas9: Big Bang In Molecular Biologymentioning

confidence: 99%

Genome Editing Using Crispr/Cas System: New Era Genetic Technology in Agriculture to Boost Crop Output

Choudhary¹,

Mushtaq²,

Singh³

et al. 2018

Eur J Exp Bio

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Genome engineering with the RNA-guided CRISPR-Cas9 system in animals and plants is revolutionizing biology. First techniques of genome editing like zinc finger nucleases and synthetic nucleases called TALENs were a starting point but turned out to be expensive, difficult to handle and timeconsuming to engineer, limiting their widespread use, particularly for large scale, high-throughput studies. Moreover, these existing technologies depending on proteins as address labels and customizing new proteins for any new change to introduce in the DNA is a cumbersome process. Of the current generation of genome editing technologies, CRISPR-Cas9 is easier to use and more efficient and can be easily targeted to almost any genomic location of choice by a short RNA guide and has been successfully applied in many organisms, including model and crop plants. Together the system has the ability to detect specific sequences of letters within the genetic code and to cut DNA at a specific point. Simultaneously with other sequence-specific nucleases, CRISPR/ Cas9 has already breach the boundaries and made genetic engineering much more versatile, efficient and easy. There really doesn't seem to be a limit in applications of CRISPR system extendable from bacteria to complex eukaryotic organisms including plants changing the pace and course of agricultural, Biomedicine and Biotechnological research in the future. This review provides an overview of recent advances in genome editing technologies in plants, and discusses how these can provide insights into current plant molecular biology research and molecular breeding technology.

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The genome editing toolbox: a spectrum of approaches for targeted modification

Cited by 32 publications

References 53 publications

Inhibition of ENaC by Endothelin-1

Inhibition of ENaC by Endothelin-1

Understanding α‐globin gene regulation and implications for the treatment of β‐thalassemia

Genome Editing Using Crispr/Cas System: New Era Genetic Technology in Agriculture to Boost Crop Output

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