Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy

Abstract

Genome editing technology is a technique for targeted genetic modifications, enabling the knockout and addition of specific DNA fragments. This technology has been widely used in various types of biomedical research, clinics and agriculture. In terms of disease research, constructing appropriate animal models is necessary. Combining reproductive technology with genome editing, many animal disease models have been generated for basic and clinical research. In addition, precisely targeted modifications allow genome editing to flourish in the field of gene therapy. Many mutations refractory to traditional gene therapy could be permanently corrected at the DNA level. Thus, genome editing is undoubtedly a promising technology for gene therapy. In this review, we mainly introduce the applications of genome editing in constructing animal disease models and gene therapies, as well as its future prospects and challenges.

Harnessing the potential of CRISPR-based platforms to advance the field of hospital medicine

“…CRISPR-based platforms have profoundly altered molecular biology and are poised to redefine the practice of medicine [48,[90][91][92]. This paper reviews some of the most promising developments in diagnostics and therapeutics that have pertinence to hospitalistsspecialists in inpatient medicinewho care for patients with a wide variety of maladies, ranging from infectious diseases to cancer to heart disease [93,94]. It is important for hospitalists to understand this emerging new technology both to ensure that it is used properly and to identify areas where it might be further deployed.…”

Rabbit Biomodels of Human Diseases Developed Using New Genomic Technologies. Crispr/Cas9 (Review)

“…Для редактирования геномов более востребованной оказалась технология РНК-направляемых эндонуклеаз -CRISPR/Cas9. Начиная с 2013 года, CRISPR-технология активно применяется для получения ГМ животных с целью их использования в качестве биомоделей для изучения физиологии и болезней человека или животных, биореакторов для получения биологически активных белков, доноров органов для трансплантации [44,57,78].…”

The Impact of CRISPR-Cas9 on Age-related Disorders: From Pathology to Therapy

“…However, this technology presents multiple drawbacks, including incomplete or insufficient knockout and off-target effects [ 11 ]. The clustered regularly interspaced palindromic repeats- (CRISPR-) associated (Cas) protein 9 (known as CRISPR-Cas9) system targets and induces site-specific DNA double-strand breaks (DSBs) directed by a single-guide RNA (sgRNA) that enables the editing of the genome by adding, removing, or altering sections of the DNA sequences in a variety of species [ 12 - 14 ]. The concept of ene-editing by a complete knockout of a gene in human cells with minimal off-targeting events represents a powerful approach to study gene function and to discern the molecular mechanisms underlying complex human diseases with the ultimate goal of improving the quality of gene therapy studies [ 15 ].…”

“…Unlike the bacterial adaptive immunity, the CRISPR-Cas9 gene-editing tool is less complex, as it requires only three components including the crRNA, trRNA, and Cas9 [ 13 , 14 , 17 , 18 , 28 , 29 ]. CRISPR-Cas9 can be used to generate an RNA-programmable method of gene editing in eukaryotic cells, allowing gene knockouts via non-homologous end joining (NHEJ), or knock-ins via homology-directed repair (HDR) [ 17 , 18 ].…”