About this Research Topic
The emerging gene editing tools mainly including meganucleases, zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR)-associated (Cas) proteins. They have been engineered to recognize and induce DNA or RNA cleavage at target sites to generate sequence editing. In recent years, some innovative CRISPR-based gene editing tools such as base editors (BE) and the prime editor (PE) have been shown to be promising technologies for highly efficient and precise targeted DNA sequence modifications. Through these gene editing tools, we could rewrite the life code to alter cell fates and even organism traits. And more importantly, somatic cell gene editing technologies have promising potentials as therapeutics for human genetic diseases, tumors, virus infectious diseases or degenerative diseases.
There are two potential ways to perform gene editing therapy for clinical applications. One is isolation of somatic cells to do gene modification in vitro and then transfer them back into organism such as sickle-cell disease and beta thalassemia. Another is derivation of gene editing tools by viral and non-viral vectors to target somatic cells in vivo, such as Duchenne muscular dystrophy, Leber congenital amaurosis type 10, autosomal recessive liver disease phenylketonuria (PKU).
Given the intense research interest, gene editing therapy could be translated to the clinic within the next 5 years. Currently, a company report has showed gene editing providing potential clinical benefit for both β-thalassemia and sickle-cell patients. In this topic, we would like to collect recent advances in gene editing therapy.
This Research Topic aims to address the following questions:
• Overview of gene editing in human diseases.
• Methods of derivation of gene editing tool in somatic cells in vivo.
• Development of new gene editing tools.
• Development of gene editing therapy for human genetic diseases, virus infectious diseases or degenerative diseases.
There are two potential ways to perform gene editing therapy for clinical applications. One is isolation of somatic cells to do gene modification in vitro and then transfer them back into organism such as sickle-cell disease and beta thalassemia. Another is derivation of gene editing tools by viral and non-viral vectors to target somatic cells in vivo, such as Duchenne muscular dystrophy, Leber congenital amaurosis type 10, autosomal recessive liver disease phenylketonuria (PKU).
Given the intense research interest, gene editing therapy could be translated to the clinic within the next 5 years. Currently, a company report has showed gene editing providing potential clinical benefit for both β-thalassemia and sickle-cell patients. In this topic, we would like to collect recent advances in gene editing therapy.
This Research Topic aims to address the following questions:
• Overview of gene editing in human diseases.
• Methods of derivation of gene editing tool in somatic cells in vivo.
• Development of new gene editing tools.
• Development of gene editing therapy for human genetic diseases, virus infectious diseases or degenerative diseases.
Keywords: Gene editing, Genetic diseases, virus infectious diseases, degenerative diseases
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