CRISPR-Cas9: These scissors can save lives one snip at a time!
CRISPR-Cas9 mediated gene editing of VEGFR2 exhibits the potential to mitigate angiogenesis-related diseases
Angiogenesis—a fundamental physiological process entailing the creation of new blood vessels—is implicated in diseases such as cancer, retinopathy, and macular degeneration. Vascular endothelial growth factor receptor 2 (VEGFR2) plays an important role in angiogenesis as it implements microvascular permeability and neovascularization in response to vascular endothelial growth factor (VEGF) binding to it. Inactivation of the receptor serves as a viable treatment for angiogenesis-related illnesses. Current treatment plans include VEGF antibodies and/or VEGFR2
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The study corroborates the ability of genome-editing to abrogate angiogenesis, however, some caveats must be addressed before considering this treatment for clinical use in humans. Safety remains a primary concern because genome editing is irreversible and any detrimental off-target mutations could impair cell function. Further, the viral delivery system could be threatening to the public, therefore, a non-viral platform can be developed to progress its clinical application. Nonetheless, the study’s findings tackle other safety aspects to support its clinical use. For example, the study reported that CRISPR/Cas9 treatment did not affect any off-target sequences. Optical examinations of the mice used in the study revealed that the intravitreal injection did not modify retinal structure and function.
Even though the CRISPR/Cas9 system is not a new phenomenon, the novelty of this research lies in the intervention of gene-editing in intraocular pathological angiogenesis. Abnormal angiogenesis is characteristic of diseases associated with blindness affecting millions of people of all ages. Since CRISPR/Cas9 technology produces a permanent genetic alteration, it
CRISPR is a new gene-modifying tool that has the potential to treat numerous medical conditions by editing genes that are responsible for certain diseases. This technology is based on the ability of bacteria to destroy the DNA of invading viruses. Studies have suggested that this new technology can be applied to human cells, although the idea of chopping up regions of the human genome can be unethical and could even be harmful. In order for the treatment to be administered to a patient, a small piece of RNA and an enzyme that makes a cut in the DNA are delivered to the cells. A biotechnology company, known as Editas Medicine, located in Cambridge, MA, is already designing treatments for conditions of the blood and the eye using CRISPR. For
During January of 2013, scientists based at the Broad Institute of MIT and Harvard were able to demonstrate fixed change in human and animal cells using CRISPR-Cas9. This marked a turning point in genetic advancement—though CRISPR was far from mastered, its potential was beginning to show.
Genome editing is a huge leap forward in science and medicine. Because of recent advances in technology, the study of genes and induced ‘point’ mutations have led to the discovery and advancement of methods previously used in order to mutate genes. The development of Clusters of Regularly Interspaced Short Palindromic Repeats (CRISPRs) and CRISPR associated system 9 protein (Cas9) technology is a hugely significant leap forward as this is a tool that could potentially be used for the research into and hopefully the treatment of a range of medical conditions that are genetically related. Cystic fibrosis (Schwank, G. et al, 2013), haemophilia and sickle cell disease are an example of some of the conditions that have the
With modern technology comes the breakthrough of the decade by altering the human genes. This altering gene invention is called CRISPR/Cas9. However, this invention in the beginning stages of altering genes, began with rats until perfection. The process began early with the embryo stages to edit the genes. With the introduction of CRISPR surrounds a lot of controversy. Some people believe editing genes is playing with the hands of God and refuse to believe in CRISPR. With the article, “Let’s Hit Pause Before Altering Humankind”, by David Baltimore believes CRISPR is a tool with no good intentions. With this information the article should not be published with being against CRISPR.
From the science community perspective, the CRISPR-Cas system could reduce or even eliminate many of the difficulties researchers face when gene editing such as cost, duration and accuracy. Prior to CRISPR-Cas, gene editing was performed in “big labs” with experts
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeat, referring to the repeating DNA sequences found in the genomes of microorganisms. CRISPR technology allows scientists to make precise changes in genes by splicing and replacing these DNA sequences with new ones. Through these changes, the biology of the cell is altered and possibly affects the health of an organism. The possibilities are endless as this offers opportunities in curing deadly diseases, modifying genes, and changing humanity as we know it. Although bioengineering has been around since the 1960s, CRISPR is significant because of the comparative low costs and the ease of the procedure to
CRISPR Cas-9 is a system that changes genes and shows a further promise for treatment for Duchenne Muscular Dystrophy (DMD). Doing this will hopefully avoid ethical dilemmas. DMD effects nearly 1 in 5,600-7,700 people between the ages of 5 and 24 for males in the U. S. CRISPR stands for “Clustered Regularly Interspaced Short Palindromic Repeats”. These are DNA segments found in microorganisms that repeat itself in open spaces. CRISPR attacks any virus that comes into your body and cuts the strand of virus on the DNA out. Researchers have tried to cure DMD in mice by getting therapy through the back of the eye, one of the ways CRISPR therapy works, which seems to work best.
The author gives a brief history of past genome editing but thoroughly explains the history and mechanism of the CRISPR technology. She elaborates on how the technology has already been used to cure diseases and speculates on its future uses and regulation.
Human gene editing has long been controversial topic; however, precise techniques that accomplish this feat have only recently been discovered. According to the Welcome Genome Campus in the UK, the most versatile and simplest technique, called CRISPR-Cas9, allows scientists to cut, alter, or add to sections of the DNA sequence of living organisms (“What Is CRISPR-Cas9?”). This astonishing technology has nearly endless applications, including the potential to eradicate genetic diseases in humans that currently have no cure. This could have vast implications for people who suffer with disease and the economy of the region in which they live, but the technology has yet to be commercialized. The
And one of the top factors in determining the viability of genetic engineering is its accuracy. In an article reported by the Interdisciplinary Center for Studies on Bioethics at the University of Chile, it was found that CRISPR has “a high frequency of off target effects…in human cells” (Rodriguez). With a high threshold for error, CRISPR does not appear to be reliable yet, at least to researchers at the University of Chile. Ethically, this raises concerns about CRISPR being an option for usage in people due to its potential risks. Genetic engineering is far more complex than any other type of medical treatment out there, so if any type of error were to occur, it would be extremely difficult to fix. Sharon Begley, a journalist for STAT news who interviewed doctors from Massachusetts General Hospital, reported that “one concern about off-target effects is that genome-editing might disable a tumor-suppressor gene or activate a cancer-causing one” (Begley). This concern was later found to be true in a separate study performed on mice by a post-doctoral colleague of UC Berkeley’s Jennifer Doudna, where CRISPR “gave rise to mutations, creating a model for human lung cancer” (Bioethics). This study provides cold-hard evidence for the journalist’s claim that even the slightest mutation can lead to cancer.
CRISPR-Cas9 gene editing is achieved by “deleting and/or inserting bases at the zygote stage of embryonic development” (Sas et al, 2017, p. 1). In theory, if gene editing occurs during the very early stages of embryonic development, “every cell in the embryo will contain this edited DNA [thus] every organ in the developing baby will contain edited DNA” (Sas et al, 2017, p. 7). However, at this stage of development, one small error could cause a significant problem. Should an unintentional removal or insertion into genes occur, the damage could lead to new diseases or deformities and “may lead to generations of unforeseen and possibly irreparable harm—such as removing someone’s protective gene for cancer” (“The Price Tag”, 2018, p. 329). In reality, gene editing may in fact introduce new genetic diseases effectively nullifying the whole point of gene
Every few years, advancements in technology alter the way scientists do their work. Recently CRISPR-Cas9, a RNA useful for working organisms in the animal kingdom has proven itself beneficial on a gene-editing platform. After performing many abortive attempts to manipulate gene function, including homologous recombination and RNA interference, scientists have finally had a breakthrough with CRISPR-Cas9.
CRISPR is versatile in that any target sequence can be modified by simply altering the gRNA sequence. In addition, multiple genes can be edited at the same time with great specificity (Cong 88-89). The convenience and accessibility of CRISPR resourced have also allowed thousands of laboratories worldwide to study CRISPR in different ways, which has broadened the horizons of its biomedical and clinical implications (Collins et al 259). Overall, the ease and simplicity of CRISPR technology has allowed for a rapid increase in the understanding of genome editing, which will allow CRISPR to revolutionize how certain conditions will be treated.
The ability to engineer biological systems and organisms has an enormous potential for applications across basic science, medicine and biotechnology. Genome editing is a group of technologies that allow scientists the ability to change an organism’s DNA, which can provide better outcomes for health and disease control compared to natural immunity and mutations. Genome editing (gene editing) allows genetic material to be added, removed or altered at particular locations in the genome. A number of gene editing technologies have emerged in recent years with one of the most versatile and precise methods of genetic manipulation being Crispr-Cas9 (Steve scott,2016). The term Crispr-cas9, (clustered regular interspaced short palindromic repeats)
CRISPR-Cas9 is a unique technology that can be used to edit the human genome (1). Compared to other techniques of editing DNA, CRISPR and Cas9 are cheaper, faster, and more accurate. CRISPR-Cas9 gives geneticists as well as medical researchers the ability to edit specific parts of the genome by the removal, adding, or altering of certain sections of DNA sequences (1). CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats and is a part of bacterial defense system (2). The two key molecules of CRISPR-Cas9 are Cas9 (an enzyme that can cut DNA at specific locations) and guide RNA (a small piece of RNA sequence that guides where the Cas9 should cut) (1). CRISPR is a crucial component