Question 1
Sickle cell anemia is a result of the mutation that takes place in the HBB gene. The gene has the blueprint that is used in the creation of protein, referred to as hemoglobin. The protein has two alpha and beta chains. For each of the single chains, the 'heme' represents the ion portion. Iron is responsible for the color of the red blood cells. It also gives the hemoglobin molecules the chance to bind and release oxygen molecules. The mutation in the HBB cells in sickle cell anemia alters one of the amino acids, which are the building blocks for the proteins present in the hemoglobin (Steinberg and Sebastiani, 2015). The mutation makes the proteins in the hemoglobin stick together and forms stiff fibers. The effect of the nucleotide change is responsible for the distortion of the shape of the red blood cells.
In a disease like Cystic fibrosis, the CFTR gene is the one that undergoes the mutation process. The mutation has taken place in a single gene. The CFTR gene consists of 1,480 amino acids. (Fertrin and Costa, 2010) When the gene is made up of all the correct amino acids, it creates a 3-D shape that is stable. Mutation makes some of the amino acids deleted, or an incorrect one can be added in the process. The process results in the malfunctioning of the nucleotide. There is the reduction of the function of the proteins leading to the creation of thick mucus that is bulk.
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Question 2
a.
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. CRISPR is a genetic sequence that is present in the genome of most prokaryotic organisms. Some of the organisms that harbor the CRISPR system include bacteria and archaea. The system is derived from the fragments of DNA make up of some of the bacteriophages that have infected the prokaryotic organism in the past (Hille and Charpentier, 2016). The CRISPR is adapted from a naturally occurring genome in the editing process taking place in bacteria. The CRISPR-Cas9 system is effective in altering the DNA sequence of the organism. It is effective in altering the organism's genomes, and they largely attribute to the traits that the organism will have.
b.
CRISPR-Cas9 is used to fix cystic fibrosis through editing. CRISPR-Cas9 is used in different series of the letter in a gene and break the DNA at that place. The method is most effective because it is less expensive. The CRISPR-Cas9 starts by locating the sequence in the DNA that has undergone mutation in cystic fibrosis. The scissors will then snip out the mutation that has taken place. The damage that has taken place attracts the mechanism of DNA repair. In the process, the broken DNA is fixed, and there is the permanent correction of the mutation process. The repair process can take place one at a time or in several mutations. That depends on how the mutation in the DNA has been arranged. The Cas9 enzyme is released by the system (Cystic Fibrosis Foundation, n.p). Through the process, it binds with the mutated CFTR proteins that have undergone mutation and cut them. The target gene is then cut off. While using the modified version of Cas9, researchers can activate the gene expression instead of cutting off the DNA gene that has undergone the mutation process. In the process, the researcher is in a position to study the functioning of the gene.
References
Cystic Fibrosis Foundation. (n.d.). Gene Editing for Cystic Fibrosis. CFF. Retrieved from https://www.cff.org/Research-into-the-Disease/Restore-CFTR-Function/Gene-Editing-for-Cystic-Fiobrosis/ . Accessed 22 June 2021.
Fertrin, K. & Costa, F. (2010). Genome Polymorphisms in sickle cell disease: Implications for clinical diversity and treatment. Expert Review Hematology, 3 (1), 443-58.
Hille, F. & Charpentier, E. (2016). CRISPR-Cas: Biology, mechanism and relevance. Philosphical transactions B, 371 (1707): 21050496.
Steinberg, M. & Sebastiani, P. (2015). Genetic Modifiers of Sickle Cell Disease. American Journal of Hematology, 87 (8), 795-803.
