With the knowledge of bioinformatics, design a practical proposal(research plan) for using the novel gene editing technology CRISPR/Cas9 to treat/cure a rare disease Sickle cell anemia.”
Requirements: As the assignment needs
just Explain Everything, and make sure there is no plagiarism. Don’t forget this is Bioinformatics Cours
Answer preview
The figure above shows that the technique involves modifying the DNA pattern to modify the gene function. The technique would involve correcting the mutation to cure the condition of sickle cell disease. Even though there are other gene-editing techniques, CRISPR/Cas9 is easily applicable, cost-effective, and highly efficient and can correct sickle cell disease mutation (Demirci et al., 2019; Savić & Schwank, 2016). On applicability, CRISPR/Cas-9 involves using a single guided RNA (SgRNAs) to recruit the target DNA sequence, an adjacent protospacer motif (PAM). Finally, a non-homologous joining repairs the associated gene, and synthetic DNA induces the desired changes. CRISPR-Cas9 may be introduced to a zygote for a permanent modification of the gene to eliminate the condition from the family lineage. Empirical evidence thus, though in the preliminary stages, identifies CRISPR-Cas9 as a treatment option
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