Genome editing is a type of genetic engineering in which DNA is inserted, deleted, modified, or replaced in the genome of a wide variety of cell types and organisms, resulting in inactivation of target genes, acquisition of novel genetic traits, and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, low off-target effect, good repeatability, and short-cycle, clustered regularly interspaced short palindromic repeats (CRISPR) technology has become the most widely used genome editing technology in molecular biology laboratories worldwide.
Genetic diseases are a leading cause of death worldwide. Although gene-supplementation therapies have been developed, they are only available for a small proportion of inherited mutations. In contrast, genome editing using CRISPR technology could provide alternative therapeutic avenues for treating a wide range of genetic diseases through targeted knockdown or correction of mutant alleles. In addition, high-throughput genetic screening based on CRISPR technology enables the exploration of genes associated with the phenotype of interest on a large scale. Therefore, novel methods of the CRISPR system have already resulted in new techniques and tools that have dramatically changed our ability to functionally annotate the genome.
CRISPR-based genome engineering technology has facilitated the rapid generation of alternative in vivo and in vitro disease models. Advances in CRISPR techniques may improve disease-modifying solutions by addressing inherited risk factors. The rapid accumulation of publicly available high-throughput genetic screening data provides a wealth of knowledge about genotype-to-phenotype relationships and a valuable resource for the systematic analysis of gene functions.
In this Research Topic, we will focus on the advances in CRISPR techniques and their applications in biomedical engineering and functional genomics studies, which include: strategies to improve the on/off-target effects of CRISPR technology; advances in CRISPR-based gene therapy in human or animal genetic diseases; application of CRISPR technology in human or animal disease modeling; applications of CRISPR screen in functional genomics; CRISPR sgRNA design tools or databases; nucleic acid or non-nucleic acid detection with CRISPR technology, etc.
Scope of the Research Topic:
• Optimization and improvement of methodologies of CRISPR technology;
• Advances in CRISPR-based gene therapy in human or animal genetic diseases;
• Application of CRISPR technology in human or animal disease modeling;
• Applications of CRISPR screen in functional genomics;
• Development of CRISPR sgRNA design tool or database;
• Nucleic acid or non-nucleic acid detection with CRISPR technology.
Genome editing is a type of genetic engineering in which DNA is inserted, deleted, modified, or replaced in the genome of a wide variety of cell types and organisms, resulting in inactivation of target genes, acquisition of novel genetic traits, and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, low off-target effect, good repeatability, and short-cycle, clustered regularly interspaced short palindromic repeats (CRISPR) technology has become the most widely used genome editing technology in molecular biology laboratories worldwide.
Genetic diseases are a leading cause of death worldwide. Although gene-supplementation therapies have been developed, they are only available for a small proportion of inherited mutations. In contrast, genome editing using CRISPR technology could provide alternative therapeutic avenues for treating a wide range of genetic diseases through targeted knockdown or correction of mutant alleles. In addition, high-throughput genetic screening based on CRISPR technology enables the exploration of genes associated with the phenotype of interest on a large scale. Therefore, novel methods of the CRISPR system have already resulted in new techniques and tools that have dramatically changed our ability to functionally annotate the genome.
CRISPR-based genome engineering technology has facilitated the rapid generation of alternative in vivo and in vitro disease models. Advances in CRISPR techniques may improve disease-modifying solutions by addressing inherited risk factors. The rapid accumulation of publicly available high-throughput genetic screening data provides a wealth of knowledge about genotype-to-phenotype relationships and a valuable resource for the systematic analysis of gene functions.
In this Research Topic, we will focus on the advances in CRISPR techniques and their applications in biomedical engineering and functional genomics studies, which include: strategies to improve the on/off-target effects of CRISPR technology; advances in CRISPR-based gene therapy in human or animal genetic diseases; application of CRISPR technology in human or animal disease modeling; applications of CRISPR screen in functional genomics; CRISPR sgRNA design tools or databases; nucleic acid or non-nucleic acid detection with CRISPR technology, etc.
Scope of the Research Topic:
• Optimization and improvement of methodologies of CRISPR technology;
• Advances in CRISPR-based gene therapy in human or animal genetic diseases;
• Application of CRISPR technology in human or animal disease modeling;
• Applications of CRISPR screen in functional genomics;
• Development of CRISPR sgRNA design tool or database;
• Nucleic acid or non-nucleic acid detection with CRISPR technology.
