The potential of CRISPR-Cas9 for treating genetic disorders

Targeted gene editing is the intentional modulation of gene expression in specific cells to treat genetic disorders. A series of programmable nuclease-based genome editing technologies have been developed in recent years. The most rapidly developing is the class of RNA-guidedendonucleases are known as Cas9 from the microbial adaptive immune system CRISPR (clustered regularly interspaced short palindromic repeats), which can be easily targeted to virtually any genomic location of choice by a short RNA guide. The CRISPR/Cas9 system is a powerful genome editing technology that can create a variety of novel therapeutics for a range of untreatable diseases in future. CRISPR-Cas systems can offer an approach for studying common human disease-such as diabetes, heart disease, schizophrenia, and autism and it can correct the causative mutation for monogenic recessive disorder due to loss-of-function mutations (such as cystic fibrosis, sickle-cell anemia, or Duchenne muscular dystrophy. It also may inactive mutated allele for dominant-negative disorders in which the affected gene is haploinsufficient (such as transthyretin-related hereditary amyloidosis or dominant forms of retinitis pigmentosum. Inactivating CCR5 receptor in lymphocytes at HIV infection and deletion of PCSK9 or angiopoietin at statin-resistant hypercholesterolemia or hyperlipidemia may provide therapeutic effect against its. Recently, the CRISPR-Cas9 system could correct the mutant Fah gene with a delivery of plasmid DNA in the liver of a mouse model of tyrosinemia. It may be possible to treat nucleotide expansion disorders, such as spinocerebellar ataxia, Huntington disease and Friedreich ataxia by NHEJ-based.

Keywords:

Targeted therapy , CRISPR-Cas9 , Gene editing