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Article by Yena Shin
Gene Editing & CRISPR
Gene editing is a powerful technique that allows for precise modifications to the DNA sequence of living organisms, effectively customizing its genetic makeup. This process involves the use of engineered enzymes, such as nucleases, to target a specific DNA sequence and cut into the DNA strands. These cuts enable the removal, replacement, as well as the insertion of DNA sequences, thus facilitating the creation of desired genetic changes.​
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Gene editing has a wide range of applications in tissue engineering and gene therapy. Within the realm of tissue engineering, gene editing is useful for creating disease models by introducing specific mutations into cells and allowing for researchers to closely study the progression of diseases and test potential treatments within a controlled environment. Furthermore, gene editing can be used to engineer functional tissues, customize cells for regenerative medicine, and enhance biomaterial integration. Gene editing can also be used in gene therapy by correcting mutations responsible for various genetic disorders such as sickle cell anemia and cystic fibrosis. This technique is additionally useful for treating cancer by modifying immune cells to better target and attack cancer cells, preventing inherited diseases by editing the genes in germline cells (sperm, eggs, or embryos), and reducing susceptibility to certain gene-linked diseases such as heart disease or diabetes. There are additionally further applications of gene editing in biotechnology, fuel, and food industries.
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Gene editing works by first identifying the target gene or DNA sequence to modify and designing the editing tool. In the case of CRISPR-Cas9, the tools include a guide RNA (gRNA), an RNA sequence that matches the target DNA sequence, and an editing protein Cas9, a protein that can cut DNA. The gRNA combines with the Cas9 protein to form the CRISPR-Cas9 complex, which is introduced into the target cells using various delivery methods such as viral. The gRNA locates and binds to the target DNA, and the Cas9 protein cuts the DNA, creating a double-stranded break. Cells naturally seek to repair the damage via non-homologous end joining (NHEJ), in which the cell’s quick repair may cause small changes (insertions or deletions) that can disable the gene, or homology-directed repair (HDR) pathways, in which a given template DNA with desired changes is used to repair the break and insert new genetic information. This process results in a successfully edited gene, whose changes can alter gene function and/or correct a mutation in a precise manner.
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There are many considerations and limitations to gene editing technologies. CRISPR editing specifically is limited by efficiency, in which not all targeted cells are edited successfully, potential off-target effects, in which CRISPR cuts DNA in unintended locations and thus causes potentially harmful side effects, and repair mechanisms, in which the cell’s natural DNA repair processes do not function as desired. In consideration to in vivo (in a living organism) gene editing, many other challenges are present. Selecting a safe and effective delivery vehicle to deliver the CRISPR-Cas9 complex to the correct cells and ensuring that the CRISPR reaches and edits only the intended cells without affecting other cells is essential, yet difficult. Furthermore, the body’s immune system may attack CRISPR components thinking they are foreign. In more controlled environments, such as in vitro (lab-based), such considerations of immune response or precise targeting within a living organism are not necessary. Gene editing also raises a lot of ethical considerations, including safety, informed consent of patients, and accessibility. More notably, editing germline cells raises concerns as any changes and unknown effects that arise in these cells would be passed onto future generations. There is much work going towards the responsible development and application of gene editing technologies to promote health and wellbeing while minimizing risk.