Gene Editing (CRISPR/Cas9)

Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR) is a genome-editing technique generated through precise editing of an organism's native genome. It has relevant applications in biotechnology, medical research and therapies, and agricultural and livestock improvement.
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Gene Editing (CRISPR/Cas9)

Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR) is a technique to achieve precise, literal cut-and-paste addition/subtraction of genomes. It relies on two major components: the Cas9 protein that can cut DNA, and the use of a guide RNA, capable of recognizing the sequence of the DNA string that can be edited.

The CRISPR/Cas9 system works by using a guide RNA (gRNA) to find the target gene, and then the Cas9 enzyme cuts the DNA at that precise location. Once the DNA is cut, the cell's own DNA repair mechanisms can either repair the cut, which can introduce errors or mutations, or the researchers can supply new genetic material to replace the removed DNA sequence.

CRISPR is more agile and accurate than previous or competing models of genetic editing. It opens up a vast range of opportunities that include editing allergens in peanuts, creating mushrooms that do not brown or even breeding genetically-engineered mosquitoes that cannot transmit diseases such as malaria.

By using CRISPR to finely control the activity of specific genes, we can better understand how organic bodies naturally carry out the same process. In terms of human system trials, researchers in China have been looking into editing human cells to create cancer-fighting white blood cells that could be injected into patients or babies vulnerable to HIV. However, despite the infinite possibilities for CRISPR, there are still crucial ethical and moral implications that arise as significant challenges and issues to be discussed, especially regarding the alteration of the human genome. We might have to decide which genes are subject to editing and which traits are prohibited from being edited in an embryo.

CRISPR CAS9 technology has become a promising alternative for crop improvement. As the world's population is expected to grow substantially in the following decades, maximizing the yield productivity without increasing the environmental impact is one of the many exciting possibilities of CRISPR. Besides, society at large seems more open to CRISPR compared to other techniques, like in the case of genetically transformed organisms, also referred to as GMOs, which have experienced intense public backlash.

Future Perspectives

Although CRISPR is currently used in very targeted manners, for example, by developing new model systems or by stimulating the effect of treatments using genetic editing, there is still a lot to be discovered in terms of side effects and reversibility. Scientists have been looking into the consequences of this gene-editing technique, especially since it may be impossible to reverse or turn off these edits.

Apart from safety risks, human genome editing poses some massive moral questions. It could offer a chance to edit harmful mutations, but for those living in poverty, genome editing could be used as one more tool for the privileged to vault ahead. Bioethics is currently debating how and where it is mandatory to trace a line between disease treatment and human enhancement to combat the creation of genetic disparities.

Taking this technology one step further, scientists are even considering bringing back extinct species, such as the wooly mammoth, with the use of the same technology. By following the same concept, companies are testing age-reversal technologies in human clinical trials, the combination of which could extend lifespans by a decade or more. One day it may be possible to eliminate the negative features of aging, erase wrinkles, and potentially wipe out age-related diseases.

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We now have the power to easily alter DNA. It could eliminate disease. It could get really out of hand.
The researchers, from the College of Veterinary Medicine, Northwest A&F University in Shaanxi, China, used a modified version of the CRISPR gene-editing technology to insert a new gene into the cow genome with no detected off target effects on the animals genetics (a common problem when creating transgenic animals using CRISPR).Image Credit: Flickr/steve p2008
Genome editing technologies enable precise modifications of DNA sequences in vivo and offer a great promise for harnessing plant genes in crop improvement. The precise manipulation of plant genomes relies on the induction of DNA double-strand breaks (DSBs) by sequence-specific nucleases (SSNs) to initiate DNA repair reactions that are based on either non-homologous end joining (NHEJ) or homology-directed repair (HDR). While complete knock-outs and loss-of-function mutations generated by NHEJ are very valuable in defining gene functions, their applications in crop improvement are somewhat limited because many agriculturally important traits are conferred by random point mutations or indels at specific loci in either the genes’ encoding or promoter regions. Therefore, genome modification through SSNs-mediated HDR for gene targeting (GT) that enables either gene replacement or knock-in will provide an unprecedented ability to facilitate plant breeding by allowing introduction of precise point mutations and new gene functions, or integration of foreign genes at specific and desired ‘safe’ harbor in a predefined manner. The emergence of three programmable SSNs such as zinc finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs), and the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein 9 (Cas9) systems has revolutionized genome modification in plants in a more controlled manner. However, while targeted mutagenesis is becoming routine in plants, the potential of GT technology has not been well realized for traits improvement in crops, mainly due to the fact that NHEJ predominates DNA repair process in somatic cells and competes with the HDR pathway, and thus HDR-mediated GT is a relative rare event in plants. Here, we review recent research findings mainly focusing on development and applications of precise GT in plants using three SSNs systems described above, and the potential mechanisms underlying HDR events in plant cells. We then address the challenges and propose future perspectives in order to facilitate the implementation of precise genome modification through SSNs-mediated GT for crop improvement in a global context.
The sterile insect technique is an area-wide pest control method that reducesagricultural pest populations by releasing mass-reared sterile insects, which thencompete for mates with wild insects. Contemporary genetics-based technologies useinsects that are homozygous for a repressible dominant lethal genetic construct ratherthan being sterilized by irradiation.
Although catalytic mechanisms in natural enzymes are well understood, achieving the diverse palette of reaction chemistries in re-engineered native proteins has proved challenging. Wholesale modification of natural enzymes is potentially compromised by their intrinsic complexity, which often obscures the underlying principles governing biocatalytic efficiency. The maquette approach can circumvent this complexity by combining a robust de novo designed chassis with a design process that avoids atomistic mimicry of natural proteins. Here, we apply this method to the construction of a highly efficient, promiscuous, and thermostable artificial enzyme that catalyzes a diverse array of substrate oxidations coupled to the reduction of H2O2. The maquette exhibits kinetics that match and even surpass those of certain natural peroxidases, retains its activity at elevated temperature and in the presence of organic solvents, and provides a simple platform for interrogating catalytic intermediates common to natural heme-containing enzymes.
CRISPR genome-editing technology shows its power
Park et al. demonstrate in vivo the efficacy of Cas9 nanocomplexes as therapeutic agents in mouse models of Alzheimer’s disease. This strategy may be applicable to the treatment of a broad range of neurological diseases.
Katie Pope Kopp went through round after round of chemotherapy and a stem cell transplant to treat her non-Hodgkin lymphoma. But nothing could beat it. "I went back to get a PET scan in May of 2020, and that's when they found that my non-Hodgkin's had blown back up, which was very disappointing," says Kopp, 64, of Parkville, Mo. She was originally diagnosed five years ago. Victor Bartolome suffered through decades of chemotherapy and a stem cell transplant, too, to keep his blood cancer at bay. Eventually, his doctors told him he had run out of options. "That was devastating. Imagine having what you think is your last hope pulled out from under you," says Bartolome, 74, of Santa Barbara, Calif. But then Kopp and Bartolome heard about something new: In the last few years, some doctors have started using the gene-editing technique CRISPR to try to modify cells of the immune system to treat cancers like theirs.
There's not enough land to feed everyone on Earth without ruining the climate, a new IPCC report shows. Gene-edited crops could help reduce agriculture's footprint.
Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to genome editing have been developed. A recent one is known as CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. The CRISPR-Cas9 system has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate, and more efficient than other existing genome editing methods.
CRISPR gene drives allow scientists to change sequences of DNA and guarantee that the resulting edited genetic trait is inherited by future generations, opening up the possibility of altering entire species forever. More than anything, the technology has led to questions: How will this new power affect humanity? What are we going to use it to change? Are we gods now? Join journalist Jennifer Kahn as she ponders these questions and shares a potentially powerful application of gene drives: the development of disease-resistant mosquitoes that could knock out malaria and Zika.
The draft rules mean that anyone who manipulates human genes in adults or embryos is responsible for adverse outcomes.
The first human trials in the US for CRISPR gene editing are officially underway. A University of Pennsylvania in Philadelphia spokesman has confirmed to NPR th...
The CRISPR-Cas9 gene editing technique is seen as a breakthrough by some and a controversy others
But the practical challenges of breeding and maintaining unconventional lab animals persist.
Development of a CRISPR/Cas9-based gene drive system in Anopheles gambiae, the main vector for the malaria parasite, paves the way for control of this pest insect.
The sterile insect technique is an environmentally-friendly insect pest control method involving the mass-rearing and sterilization, using radiation, of a target pest, followed by the systematic area-wide release of the sterile males by air over defined areas, where they mate with wild females resulting in no offspring and a declining pest population.
Gene editing offers dramatic advances in speed, scope and scale of genetic improvement. It also offers an opportunity for more nuanced GMO governance.
For a rat, there are worse ways to die. Until now, options for pest control ranged from a slow death by poison, a faster death by being trapped, to a deeply un
Where did it come from? How do organisms use it without self-destructing? And what else can it do?
A powerful new biotech tool could let us hack evolution, edit entire species, or eliminate malaria. But scientists are urging caution.
A study has found that CRISPR can delete large chunks of DNA, suggesting it could cause cancer if used to treat diseases by editing many cells in the body
Medicine is at a turning point, on the cusp of major change as disruptive technologies such as gene, RNA, and cell therapies enable scientists to approach diseases in new ways. The swiftness of this change is being driven by innovations such as CRISPR gene editing, which makes it possible to correct errors in DNA with relative ease.
We model the release of insects carrying an allele at multiple loci that shifts sex ratios in favor of males. We model two approaches to sex ratio alteration. In the first (denoted SD), meiotic segregation (or sperm fertility) is distorted in favor of gametes carrying the male-determining genetic element (e.g., Y-chromosome). It is assumed that any male carrying at least one copy of the SD allele produces only genotypically male offspring. In the second approach (denoted PM), the inserted allele alters sex ratio by causing genetically female individuals to become phenotypically male. It is assumed that any insect carrying at least one copy of the PM allele is phenotypically male. Both approaches reduce future population growth by reducing the number of phenotypic females. The models allow variation in the number of loci used in the release, the size of the release, and the negative fitness effect caused by insertion of each sex ratio altering allele. We show that such releases may be at least 2 orders of magnitude more effective than sterile male releases (SIT) in terms of numbers of surviving insects. For example, a single SD release with two released insects for every wild insect and a 5% fitness cost per inserted allele could reduce the target population to 1/1000th of the no-release population size, whereas a similar-sized SIT release would only reduce the population to one-fifth of its original size. We also compare these two sex ratio alteration approaches to a female-killing (FK) system and the sterile male technique when there are repeated releases over a number of generations. In these comparisons, the SD approach is the most efficient with equivalent pest suppression achieved by release of approximately 1 SD, 1.5-20 PM, 2-70 FK, and 16-3,000 SIT insects, depending on conditions. We also calculate the optimal number of SD and PM allele insertions to be used under various conditions, assuming that there is an additional genetic load incurred for each allelic insertion.
CRISPR is a gene editing technique used to increase food production and tackle disease. But it has also heightened concerns around genetic modification.
A series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across the biological sciences. The discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) proteins has expanded the applications of genetic research in thousands of labs across the globe and is redefining our approach to gene therapy. Traditional gene therapy has raised some concerns, as its reliance on viral vector delivery of therapeutic transgenes can cause both insertional oncogenesis and immunogenic toxicity. By obliviating some of the concerns raised by traditional gene therapy, CRISPR technology provides a relatively simple and efficient alternative for site-specific gene editing. Although it has apparent advantages, CRISPR/Cas9 brings its own set of limitations which must be addressed for safe and efficient clinical translation. This review focuses on the evolution of gene therapy and the role of CRISPR in shifting the gene therapy paradigm. We review the emerging data of recent gene therapy trials and consider the best strategy to move forward with this powerful but still relatively new technology.
Report calls current regulations inadequate and emphasizes the need for early public involvement and international coordination for new technology
New research suggests that a controversial gene-editing experiment to make children resistant to HIV may also have enhanced their ability to learn and form memories.
A media article describing how researchers use CRISPR to edit tomato genomes and increase crop yield.
It’s only been seven years since scientists first learned how to precisely and reliably splice the human genome using a tool called CRISPR, making it possible to think about snipping out disease-causing mutations and actually cure, once and for all, genetic diseases ranging from sickle cell anemia to certain types of cancer and even blindness.

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