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CRISPR/Cas9-mediated Genome Editing: In VivoReview

Narges Lotfalizadeh1, Soheil Sadr1, Pouria Ahmadi Simab2, Ashkan Hajjafari3Hassan Borji4, and Zeynab Bayat5,*

1Department of Clinical Sciences, Faculty of Veterinary Medicine, Ferdowsi University of Mashhad, Mashhad, Iran

2Department of Clinical Sciences, Faculty of Veterinary Medicine, Sanandaj Branch, Islamic Azad University, Sanandaj, Iran

3Department of Pathobiology, Faculty of Veterinary Medicine, Islamic Azad University, Science and Research Branch, Tehran, Iran

4Department of Pathobiology, Faculty of Veterinary Medicine, Ferdowsi University of Mashhad, Mashhad, Iran

5Department of Biology, Faculty of Sciences, Shahid Bahonar University of Kerman, Kerman, Iran



The CRISPR/Cas9 system has been a game-changer in genetics and biotechnology. This study aimedto investigate the existing in vivouses and its potential ingene function and biological processes usinganimal models. With its remarkable precision and accuracy, researchers can now easily edit specific genes within cells and organisms. This technology has opened up new avenues for studying genetic diseases and developing therapies to treat them. One of the most significant advantages of the CRISPR/Cas9 system is its ability to create precise cellular and animal models of human diseases. This allows researchers to investigate the role of genetics in disease development and to develop more effective therapies. For example, the system can correct genetic mutations that cause cystic fibrosis or sickle cell anemia. The therapeutic potential of CRISPR/Cas9 is enormous, especially in gene therapy. By correcting specific genetic mutations, the system can potentially treat human diseases that are currently untreatable with conventional therapies. However, some challenges still need to be addressed before this technology can be used in clinical settings. Despite these challenges, the potential of CRISPR/Cas9 to revolutionize the field of genetics and biotechnology cannot be overstated. Ultimately, this technology has the potential to transform medicine by providing new therapies for a wide range of genetic diseases.

1. Introduction:

Genome editing is a type of genetic modification that involves manipulating DNA at the level of individual bases1. It has revolutionized biomedical research by holding great potential for treating and preventing various human genetic disorders. However, the most effective genome-editing tool needs to be highly specific in modifying genomic sequences while minimizing off-target effects2. Initially, genome-editing techniques involvesreplacing small genome sectionswith external donor DNA sequences using the homologous recombination repair pathway in yeast and mammalian cells3. Similarly, mouse embryonic stem cells were also used to create mice with specific genotypes4. However, these techniques have limitations, such as low editing efficiency and unwanted genome modifications that occur at random sites rather than at the intended location 5.To overcome these limitations, scientists have developed Meganucleases, which are endonucleases that cut specific DNA sequences to stimulate homology-directed repair (HDR)6. This approach introducessite-specific double-stran.

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