The Application of CRISPR/Cas9 for the Treatment of Retinal Diseases
نویسندگان
چکیده
The CRISPR/Cas9 system of genome editing has revolutionized molecular biology, offering a simple, and relatively inexpensive method of creating precise DNA edits. It has potential application in gene therapy treatment of retinal diseases providing targeted disruption, alteration, or transcriptional regulation of pathogenic genes. In vivo studies have demonstrated therapeutic benefit for a variety of diseases. Despite this, there are many challenges to clinical use of CRISPR/Cas9, including editing efficiency, off-target effects, and disease heterogeneity. This review details the mechanisms of the CRISPR/Cas9 system and the treatment strategies that can be applied to retinal diseases. It gives an overview of in vivo studies published to date and discusses the challenges and potential solutions to the wide-scale clinical use of CRISPR/Cas9 as a therapeutic intervention.
منابع مشابه
The application and mechanism of CRISPR-Cas systems in the treatment of infectious diseases
Infectious diseases remain a global threat with many people annually contracting the epidemic diseases. Improved understanding of the pathogenesis of bacteria, viruses, fungi, and parasites, along with rapid diagnosis and treatment of human infections are essential to improving infectious diseases outcomes worldwide. In many genomic loci in bacteria and archea, termed Clustered Regularly Inters...
متن کاملDeveloping oncolytic Herpes simplex virus type 1 through UL39 knockout by CRISPR-Cas9
Objective(s): Oncolytic Herpes simplex virus type 1 (HSV-1) has emerged as a promising strategy for cancer therapy. However, development of novel oncolytic mutants has remained a major challenge owing to low efficiency of conventional genome editing methods. Recently, CRISPR-Cas9 has revolutionized genome editing.Materials and Methods: I...
متن کاملفناوری ویرایش ژن کریسپر ـ کَس 9 از منظر حقوق مالکیت فکری و ایمنی زیستی
In recent years, inexpensive and fruitful gene editing techniques such as CRISPR-Cas9 and NaAgo have revolutionized the biotechnology industry. Genetically edited organisms, gene therapy, treatment of diseases such as AIDS and editing human cells are some of the marvelous applications of such technologies. Using such technologies in large scale or granting exclusive rights on their products or ...
متن کاملThe new genomic editing system (CRISPR)
Over the past decades, progression in genetic element manipulation, and consequently, the treatment of diseases has been remarkable. It is worth noting that these genetic manipulations perform at different levels, including DNA and RNA. The earlier genomic editing techniques, including MN, ZFN , TALEN , performing their functions by creating double-stranded breaks (DSBs), and after breakage, th...
متن کاملTransfusion Related Adverse Effects on Beta-Thalassemia Major and New Therapeutic Approaches: A Review Study
Thalassemia is one of the most common genetic disorders, worldwide.Beta-Thalassemia Major (BTM) is the most severe type, which reduces lifeexpectancy and quality of life. In this study, we searched the related keywords to subject from 1996-2019 in the Medline and Web of Science databases, therefore found 250 articles. Moreover, we categorized them into the studies on blood transfusions in...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
عنوان ژورنال:
دوره 90 شماره
صفحات -
تاریخ انتشار 2017