Introduction:
An ongoing investigation has indicated that CRISPR can be utilized as a generative method that can treat Duchenne muscular dystrophy. Because of an examination in mice, it could be created as a remedial choice for humans Duchenne muscular dystrophy is caused by a defective gene for dystrophin. Duchenne muscular dystrophy occurs in about 1 out of every 3,600 males commonly between 3 to 6 years. As this is an inherited disorder, risks include a family history of Duchenne muscular dystrophy.
In 2016, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke, incorporated one of the first successful uses of CRISPR to treat an animal model of the genetic disease with a technique that can potentially be used for human treatment. Gersbach effectively showed in a mouse model that CRISPR can recover muscle suffering the Duchenne muscular dystrophy (DMD). His methodology utilizes CRISPR to cut out dystrophin exons around the genetic mutation, leaving the body's normal DNA fix framework to repair the remaining gene back together to make a shortened, however useful adaptation of the dystrophin gene.
Other studies have reported that the immune system within mice can stimulate a response to CRISPR, which could possibly intervene with the benefit of CRISPR therapies within the human body. Multiple groups have also reported that some people have developed immunity to CRISPR proteins, likely due to previous exposure to the bacterial host, however, where this would occur is unknown.
Recent years have witnessed the development of tools which actively assist researchers in performing CRISPR-Cas9 experiment optimally. These tools specifically aim to maximize on-target while also minimizing potential off-target effects by analysing the features of the target site, within the Dystrophin gene, causing the inherent disorder.
About the biotechnology:
CRISPR (clusters of regularly interspaced short palindromic repeats) is a simple however powerful tool which is able to edit genomes. It has recently allowed researchers to alter DNA sequences and modify gene function. CRISPRs are specialized stretches of DNA with two major characteristics: the presence of nucleotide repeats and spacers.
The CRISPR model allows for targeted genome editing of DNA sequences and strands. The biotechnology is focused to the DNA through contact with a guide RNA (gRNA) molecule, which then binds to the targeted DNA block through base complementarity and then allows precise DNA cleavage. This cleavage is then repaired through multiple pathways, which can be modified to the required outcomes.
Forcing a gene to be inoperative (knockout) can then be attained via error-prone repair through the Non-homologous end Joining pathway, which has the ability to introduce mutations and disrupt gene function. Targeted incorporated of a sequence can be achieved via the Homology Directed Repair pathway, which uses a provided DNA template to repair the cleavage of the Dystrophin gene.
Activation or repression of a gene can be accomplished through focusing catalytically inert Cas9 combined to a transcription activator or repressor to the promoter. All of these methods require the precise and effective focusing of the CRISR framework to the ideal location. The achievement of an analysis utilizing the CRISPR framework consequently depends on the right recognizable proof of the ideal objective site and ensuing structure of the complimentary gRNA).
Contributions of the use of biotechnology (how could it be applied in the context of your choice and comparison with current methods and the effect of biodiversity)
Steroid medications can slow the loss muscle strength, however, there is yet no cure of Duchenne muscular dystrophy. Treatment intends to control symptoms to improve quality of life. Other treatments include medications which include Corticosteroids, such as prednisone and deflazacort, which can help muscle strength and delay the progression of certain types of muscular dystrophy.
Many forms of therapy and assistive devices can improve the quality and in some cases the lifetime of individuals diagnosed with muscular dystrophy, examples include, range-of-motion and stretching exercises. Muscular dystrophy can restrict the flexibility and mobility of joints, these Limbs often draw inward and become fixed in that position. Range-of-motion exercises can help to keep joints. However, unlike a CRISPR used approach for treatment there is no guaranteed results and many methods have side effect symptoms.
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There have also been past gene-editing technologies, for example, gene replacement. Gene replacement therapy is the technique of recognizing a faulty gene, then applying a piece of DNA in its correct form though a viral vector to the gene, in turn being able to override the identified faulty gene with the now correct copy. Gene replacement seeks to alter genes to correct genetic defects and thus prevent or cure genetic diseases. Three various gene therapy methods can be utilized to restore dystrophin expression. These are gene repair, exon skipping, and dystrophin gene replacement.
What was previously attempted with gene editing was to manipulate genetic information in blocks, basically in big pieces. The power of CRISPR technology. Thus, the precision of CRISPR is greater than other biotechnologies. This allows scientist to make changes in DNA of actual genes where they can “Turn off” harmful genes or they can potentially repair genes that have got mutations within them where the code is written incorrectly.
A successful gRNA must maximize on-target activity while also minimizing potential off-target effects. Adjusting both requirements can be a combinatorial task and thus, a significant effort in the ongoing years have been aimed at creating computational tools to aid in the design of gRNAs. These tools are aimed to help researchers in the selection of desired target sites by helping them exclude unwanted targets based on anticipated low productivity or a high potential for off-target impacts.
Researchers have recently successfully demonstrated in a mouse model, how CRISPR can regenerate muscle suffering from Duchene muscular dystrophy. To treat muscular dystrophy, a viral delivery system could provide patient cells with the instructions to make the Cas9 protein, as well as the RNAs that target specific regions of DNA. This has led to the possibility of CRISPR to treat the large population with the condition.
Risks and benefits (consider bioethics, and social impacts)
CRISPR can be an extremely effective tool in editing genes and to potentially treat Duchene muscular dystrophy. However, it still must be confirmed as an effective technique. This has caused various researchers to strive for improvements in this area, to make the process increasingly precise and effective, for future potential benefits.
CRISPR technology is also simple and cost-efficient unlike other gene-editing techniques. This technology can also be used to analyse the interaction of genes and the relationship between genetic differences and expression (phenotype). If CRISPR is successful in treating Duchene muscular dystrophy, it will inevitably lead to a significant decrease in the disease, as it is inherited. This will in turn lead to a significant decrease in investment put into research to find a cure for the disease, thus providing more funding into other illnesses.
Within previous months, Increased concerns have been raised regarding CRISPR. Various studies have suggested that CRISPR technology being used on humans could possibly cause cells within human bodies to lose their cancer-fighting ability. Thus, doing more damage to genes than originally thought. This raises many concerns about whether CRISPR should be used in humans as though it may cure Muscular Duchenne Dystrophy for children affected at young ages it may cause further health issues throughout their life which may be more life-threatening than Duchenne Muscular Dystrophy.
Future directions of the biotechnology
The currently available tools of CRISPR cannot just simply predict the success of an experiment as prediction accuracy depends on various factors including the accuracy of the approximations of tested models and how similar the experimental setup matches the data the model was based off of. Some patients were reported to have pre-existing immunity against Cas9, as they were once exposed to Cas9-carrying bacteria.
Fusing environmental data in future predictive models will help improve the precision of data collected through conducted experiments and this is essential if the technology is confirmed to be successful where it can be applied within the clinics and hospitals for children. Such modeling will also allow for the selective targeting of individual muscles, which contain the defective gene, causing illnesses in children.
Incorporation of chromatin environments would further likely improve off-target predictions of ways to treat the illness, as it will be more suitable to assisting accessibility of Chromatins. Other than chromatin data, potential off-target pipelines should also concentrate on including variant data. A recent study demonstrated that the differences between individuals have dramatically affected the off-target ability, with point mutations creating and destroying potential off-target sites.
Future models may also not only simply predict the success of CRISPR editing within the Dystrophin gene, but additionally the increase the success of potential results. By targeting sites with microhomology and exploiting the microhomology-mediated repair pathway, researchers may be able to delete specific DNA segments and subsequently control the results of CRISPR-Cas9 editing within these children, thus being a potential remedy to the illness.
Conclusion
As researchers' understanding improves regarding CRISPR within Duchene Muscular Dystrophy, there will be larger incorporation into new features into predictive models to increase their accuracy. Gaining an informed understanding through various research and experiments conducted are essential for applying CRISPR-Cas9 in clinical applications, where individuals are left vulnerable. Until CRISPR technology is capable of being used to treat Duchenne Muscular Dystrophy, scientists must sustain this understanding to ensure this cure is effective for human beings.
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