An Examination of the CRISPR Cas9 Treatment for Cystic Fibrosis

Cystic fibrosis is a hereditary disease caused by a mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene on chromosome 7 which damages the lungs and digestive systems. As scientists have aimed to cure and prevent cystic fibrosis, distinctive technology CRISPR Cas9 has been introduced which allows scientists to make changes to DNA in cells that could cure genetic disorders. The development of inquiry question ‘does CRISPR Cass have the potential to accurately prevent and cure cystic fibrosis? ‘, allows the report to contain in—depth research regarding the effectiveness of CRISPR Cass technology as a potential treatment for cystic fibrosis.

The following report will outline genetic disease. cystic fibrosis. as well as an evaluation of the risks and benifits of genetic technology CRISPR Cas9 and social, economic and considerations surrounding its usage.

The Genetic Disease: Cystic Fibrosis Cystic fibrosis (CF) is a gradual genetic disease and complications are initiated through the CFTR protein channel. found throughout the respiratory tract. Affected individuals have elevated sweat electrolyte levels, pancreatic insufficiencies and lung infections.

These signs are observed in most patients but not all since the severity of the disease can range widely amongst individuals. Inllammations and infections of the sinuses affect epithelium found in the upper respiratory tract and digestive functions are altered as food, specifically fats and proteins. are not digested properly as mucus blocks digestive enzymes. As the disease is a germ-line mutation, age of onset for cystic fibrosis is inherited through individuals if the offspring‘s parents have 1 defective copy of the gene.

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Recent studies have shown a higher understanding of the underlying causes of cystic fibrosis at a genetic level as more than 1.500 genetic mutations have been linked to the onset of this disease.

However the most relevant mutation found in 66% of affected patients is the deletion of the phenylalanine in position 508 on the CFTR gene. This inherited disease mainly infects the lungs, pancreas, liver. intestine and sinuses. The most prominent organ affected are the lungs because excess mucus causes infection lung damage due to trapped bacteria. The mutated CFTR gene causing cystic fibrosis becomes the polypeptide from this gene and acts as an outlet across the membrane of cells that produces sweat. saliva, mucus and digestive enzymes. The genotype of cystic fibrosis derive in a build-up of mucus is due to a disturbance in the water/salt balance in the epithelial cells. The phenotype of this disease causes infections and eventually permanent lung damage as poor nutrition restricts diets. Cystic fibrosis is an autosomal recessive disorder, meaning there is a mutation in both copies of the gene in a cell.

Inheritance patterns are through parents of the offspring with an autosomal recessive condition, each carrying a singular copy of the mutated gene, however, does not show sign and symptoms of the condition. There are over 70.000 individuals worldwide who are affected by cystic fibrosis and 75% of affected individuals are diagnosed from the age of two. Cystic fibrosis birth prevalence in Australia was reported to affect 1 out of 3700 people. Additionally, ten disease is most common in European descent as 1 in 25 Australians carry the genetic mutation. Although cystic fibrosis has no successful treatment at present and management strategies concentrate on reducing the severity of the symptoms as research is currently being undertaken into the use of gene editing to treat this disease. With this advanced knowledge of causes to cystic fibrosis, several treatments have been proposed with the intent of these treatments being to prevent and control infections in the lungs by removing mucus and to prevent blockages in the intestines. Treatment of this cystic fibrosis include antibiotics for infections of the airways, chest physical therapy, and other specific medications.

Cystic fibrosis has no effective treatment regarding prevention and the quality of life of affected individuals is compromised and there is a continuing financial burden on the individual, their family and the health system. The scientific method currently being used to treat cystic fibrosis is CRISPR CasQ, (Clustered regularly interspaced short pelargonic repeats). CRISPR consist of two components; the Cas9 protein which cuts DNA and the Guide RNA that can recognise the sequence of DNA to be edited. Scientistic utilise the CRISPR program system by inserting an affected individual own guide RNA. guiding the enzyme to the gene preferred to be edited, modified, delete or inserting new sequences CRISPR Cas9 is currently an experimental method for treating cystic fibrosis. In cystic fibrosis, the cilium is prevented from clearing mucus as it is pressed against cilia.

The cause is due to an abnormal CFTR protein created from genetic mutation inside the cell nucleus. Gene editing technologies such as CRlSPR CasQ repairs abnormal DNA sequence by cutting out and replacing defective parts of DNA. Repair systems have three components; a guide RNA seeks to bind to a specific location in the DNA, the Ca59 nucleus enzyme cuts genetic sequence at the binding site, and repairs template containing the correct genetic sequence of the gene. Once these components are introduced into a cell. the guide RNA binds and activates the CasS protein, which can search the cell‘s DNA for potential targets for Cas9 to unwind a section of DNA to detect if the guide RNA sequence matches. Once strand is repaired. the template falls away and other strands of DNA uses repaired stands to fill in remaining gaps and creating corrected gene sequence.

The cells then use the corrected DNA strand to produce a functional CFTR protein that allows proper flow of salt and fluids through the cell. Evaluation of Inquiry Question and Genetic Technology: Through evaluating my inquiry question ‘does CRISPR C059 have the potential to accurately prevent and cure cystic fibrosis?’ reveals the advantages and validity, however there as disadvantages and implications of utilising CRISPR in curing cystic fibrosis, CRISPR is efficient and simple as it can directly target an embryo, reducing the time required to modify target genes compared to other gene targeting technologies. Limitations of CRISPR include the effects of off-target as they generate unfavourable mutations at random sites, resulting in genomic instability and delaying clinical procedures. Although CRISPR Cas9 has been chosen as the desired method of genome editing, social and ethical issues arise concerning its use.

Ecological disequilibrium has an impact on biodiversity as it interferes with biological individuals which increase off- target mutations through each generation and may cause ecological damage or affect human health. Although CRISPR is inexpensive. there is a lack of economic ability as CRISPR is exclusive to the wealthy and gives the demographic an unfair advantage. Social implications arise as parents are influenced through societies values and can advancing offspring traits through gene editing such as an unfair advantage of enhanced performance in athletics. These implications result in the technology being used for non-medical and unimportant uses. The validity of CRISPR is exemplified though the targeted deep sequencing in human cells as it reduced the additional energy released by the RNA—DNA complementarily, reducing Ca59 nuclease to be specific on cleavage procedures, CRISPR gene editing has successfully helped cure similar cystic fibrosis mutations with a targeting efficiency of 722%.

Although the disease currently has no cure available, CRISPR CasQ can treat the genetic cause as it targets particular mutations The UniTrento/KU Leuven research term amended the CRISPR Cas9 system into permanently editing two types of mutations causing cystic fibrosis using the Splice Fix technique which fixes and restores protein in the gene. CRISPR Cas9 is considered to be greater in comparison to other gene-editing technologies due to its customisable qualities as it targets specific DNA and does not need to be paired with different enzymes to remove pieces of DNA as it secrets its own CasS(Mul\/ihill et al., 2020) Additionally. CRISPR is inexpensive compared to previous techniques such as transcription activators-like effective nucleases (TALENS).

Furthermore CRISPR simply matches with the guide RNA through thousands of sequences are introduced. targeting numerous genes and DNA sequences at the same time, As CRISPR has been effective in helping to treat cystic fibrosis, gene therapy has been studied as a potential treatment for genetic diseases Gene therapy focuses on insening a corrected CFTR gene into a cell, providing an operative copy of the regular CFTR protein. Gene therapy works with any individual with cystic fibrosis and can be utilised by scientist as future prevention of cystic fibrosis Conclusion and future implications Scientists anticipate utilising CRISPR Gas?) to develop critical advances in patient care and the possibility of curing the inheritable disease cystic fibrosis.

Future studies are required for scientists to gain a profound understanding of the mechanisms underlying CRISPR CasQ gene editing for suitable clinical applications. CRISPR is anticipated to commence major improvements and beneficial applications which will be accomplished through severe supervision policies to minimise problems. Throughout the research of inquiry question ‘daes CRISPR Casf) have the potential to accurately prevent and cure cystic fibmsis?’, allowed the report to understand and investigate the linguistics of CRISPR and its influence on the mutation cystic fibrosis CRISPR technology is a favourable tool for gene editing which offers researchers the opportunity to modify gene function. Despite the technology unable to cure and prevent cystic fibrosis, its approach provides an effective tool to repair the CFTR mutation through gene editing.