CRISPR-Cas9: Changing Genes

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

CRISPR has been garnishing a lot of media attention recently and it is not just popular among the scientific community but also the general public. Several online news outlets and scientific journals have been talking about the significance CRISPR-Cas could have for the field of genetics and science as a whole. I even came across a Youtube video from The Verge, a tech channel that normally does reviews on new smartphones and laptops talking about CRISPR [15]. So why is CRISPR gaining so much attention both from the scientific community and the general public? The answer lies in the potential this technology possesses.

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

Due to CRISPR-Cas9’s low cost, global scientific research on genetic editing has flourished. The power of CRISPR “is so easily accessible by labs — you don 't need a very expensive piece of equipment and people don 't need to get many years of training to do this” (Ledford). Because of this, CRISPR technology has spread to labs across the globe. There are two main economic

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)

Biotechnology prompts a fear that humanity is gaining too much control over the choice of human evolution and destiny (McLean 1). CRISPR, “…the new fast, flexible, cheap way to manipulate the genetic code of life…” (Achenbach 1) exploits from the natural process used by ordinary bacteria to fend off against intrusive viruses while Cas (CRISPR-associated proteins), a set of enzymes, precisely snips DNA. CRISPR makes it simple to alter specific genes, resulting in many scientists beginning to use this new form of biotech for many of their experiments. Consequently, the editing of the

However one of the most prominent features of CRISPR’s future, that has people on the edge of their seat is that perhaps some day CRISPR could be used in order to create

CRISPR-Cas9, a genome editing instrument, moves to change the field of biology forever. CRISPR was first observed as an innate defense mechanism used by bacteria. After years of development, scientists have been able to construct their own RNA that guides the CRISPR-Cas9. This allows them to control the behavior of the CRISPR-Cas9. What this could mean for the future is overwhelming.

The human species have the extraordinary characteristic that has given us the ability to flourish all over the world. Humans are adaptive; refashioning our surroundings and passing this information down to the next generation to be built upon, has lead to a thriving species. Now, instead of modifying solely our outside environment, it is possible to also engineer our own genetic makeup. This genetic engineering is known as CRISPR. The system was originally discovered in bacteria as a protection mechanism against unknown pathogens; however, after being repurposed into a more generally practical technology, the genetic nature of essentially any species can be revised. This modernizing genomic engineering system has equipped scientists with the ability to precisely modify the DNA of almost any organism, including humans. CRISPR finds the specific strand of DNA that is unfavorable and replaces it

CRISPR-Cas9 is not an artificial construction; bacteria use it as a form of adaptive anti-viral immunity. When infected with a pathogen, bacteria that has this system retain a portion of the infecting virus in their chromosomal DNA. The cell then transcribes those sequences (called CRISPRs, or “clustered regularly interspaced short palindromic repeats”) and processes them into short RNAs called crRNAs. The crRNA guides the Cas9 nuclease to its target (normally, an invading viral nucleic acid) and cuts the foreign nucleic acid, rendering the virus useless in the process.

A gene editing method called CRISPR, Science’s 2015 Breakthrough of the Year, is an easy and inexpensive way to cause the “DNA-cutting enzyme” to cut out specific parts of the DNA strand, therefore changing the genetic composition of an organism. Another way to use the CRISPR is to use it to deliver regulatory factors to successfully turn genes on or off and any level in between, which can help save lives by turning off deadly genes. CRISPR is currently being used for biomedical purposes, such as repairing genetic defects or creating tissue-based treatments. This genetic editing method could be used and is being used to change the very animals and plants that we know today by creating allergen-free peanuts or virus resisting pigs.

The CRISPR-Cas9 research and recent advances in animal models are showing promise to treat lots of human genetic diseases in the future. In a mainstream science article published by IFLScience!, they highlight research being done at Salk University that is working on using CRISPR-Cas9 and HITI technology to target non-dividing cells of the eye to help improve vision in rats with retinitis pigmentosa.3 The article briefly mentions what the CRISPR-Cas9 system is, stating that CRISPR is a method that snips strands of DNA and replaces those with new genetic material.3 Other than the effects of CRISPR research, and what it could mean for the future of genetic diseases, IFLScience! fails to mention the complexity of how the complex works, the engineered specificity of it, and the problems with current delivery.

Neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease(PD) and Huntington’s disease (HD), have some common characteristics like the age-dependent gathering of misfolded proteins and selective neurodegeneration. Animal models are highly valued and have been used widely to study neurological disorders and to find therapeutic aims for them. Because genetic DNA mutations can cause several neurodegenerative diseases, the ability of CRISPR/Cas9 to directly target any gene in one or two alleles of the embryonic genome opens a new route for using this new technology producing animal models of neurodegenerative diseases. The traditional gene targeting technology made it difficult to establish large animal models of human diseases due to the lack of embryonic stem cell lines. Since large animals are closer to humans, their disease models may more faithfully mimic the clinical symptoms of patients and are important for exploring the mechanisms and treatment of both neuropsychiatric disorders and age- related neurodegenerative diseases. CRISPR/Cas9 was found to functionally disrupt the dystrophin gene in founder monkeys and causes the same muscle atrophy phenotype seen in patients. Thus, when both alleles are mutated by CIRSPR/Cas9, the complete loss of Parkin or Pink1will mimic the genetic mutations in PD patients. Also, because CRISPR/Cas9 can target multiple genes in the same cells,