Guide to Figure out The Application Prospects of CRISPR Gene Editing

In a recent study, researchers at the Massachusetts Institute of Technology's Bode Institute and Harvard University programmed a CRISPR-related enzyme to target three different single-stranded RNA viruses in human embryonic kidney cells ( as well as human lung cancer cells and dog kidney cells) are grown in vitro and minced so that they are largely incapable of infecting other cells. If further experiments indicate that the process is effective for living animals, it may eventually help to identify new antiviral therapies for diseases such as human Ebola or Zika disease.

CRISPR is a gene editing technology, a "molecular scissors" that allows scientists to "cut" and "paste" genes into DNA and perform gene editing faster and more accurately. Based on a targeted DNA destruction defense system originally discovered in certain prokaryotes, CRISPR has revolutionized medical science by introducing easy-to-use genome modification methods.

CRISPR plays an important role in gene expression regulation. Cas9 is fused to a well-characterized transcriptional regulatory domain. By using inactivated dCas9 protein, the complex can be targeted to a specific locus without the need to cleave or alter genomic DNA. After binding of Cas9 to the target DNA sequence, the fused transcriptional regulatory domain can be recruited to inhibit or activate effectors to modify gene expression.

As for the role of CRISPR in drug research, CRISPR/Cas makes it easier to create cells that accurately mimic disease and the entire animal model system, enabling scientists to more accurately discover new drugs and verify drug safety and efficacy, ensuring that that these models can be better represented in clinical trials. The CRISPR library detects live cells with specific conditions, researchers can identify genes and proteins that cause or prevent disease with this system, thereby identifying potential drug targets.

Over the past decade, CRISPR-based technologies have been used in a variety of tasks, from removing genes that cause disease to destroying drug-resistant superbugs to creating molecular recording devices. Cells are the most commonly used tools in the laboratory. The combination of the CRISPR system and the stable strain construction allows researchers to obtain passaged cells that can be stably edited in less time. Besides, CRISPR has been widely used in the construction of animal models such as cancer, nervous system diseases and genetic diseases, which not only greatly accelerated the construction time of model animals, but also better simulated human complex diseases with its diverse genetic editing forms. Such models can be used for gene/protein function studies, disease pathology studies, drug screening, etc.

Compared to other gene therapy strategies, CRISPR genome editing is considered faster, cheaper, and potentially safer. Autologous CRISPR cell therapy using genome editing to correct mutations in the patient's own cells is promising in bypassing the exclusion problems that exist in donor-matched transplantation therapies. The CRISPR genome editing is especially useful for diseases that can be resolved by modifying cells that can be easily removed from the patient, allowing for additional screening to ensure that off-target genome modifications will not occur during genome editing.

Standardized gene editing, even the simplest modification of cell lines, requires the design and synthesis of sgRNA, cell culture, cell transformation, variant screening and identification. From an academic point of view, there are currently two main intervention directions for CRISPR gene editing. One is for disease caused by a congenital genetic defect that humans are currently unable to treat with normal medical treatment. Through CRISPR technology, changes in the regulation of human genes to achieve therapeutic goals, there is no obvious controversy in this research direction. The other is prophylactic gene editing, which eliminates the possibility of future illness by genetically editing the embryo. There is a big controversy in this research direction. The main controversy is that the argumentation of this medical method is not sufficient. In the aspects of comparison with drug means, necessity demonstration and rationality of heritability change, it is still necessary to continuously supplement and improve the evidence.

Despite the controversy, CRISPR is still one of the most powerful genetic editing tools in the predicted future. Most scientists believe that if one day this technology is applied on a large scale, it must be used in the treatment of genetic diseases that humans cannot cure through normal medical treatment.

Although CRISPR technology is much more improved in terms of ease of use, effectiveness, and cost than other gene editing techniques. It has the potential to make revolutionary advances in disease investigation, prevention and treatment. However, the efficiency of CRISPR gene editing technology still needs to be further stabilized and improved. It is necessary to have relevant data to carry out reasonable medical arguments. https://www.creative-biogene.com/crispr-cas9/solution.html