Can genome editing transform ocular disease treatment? Nature UCIrvine ocular oculardisease eyedisease genetics genomeediting geneediting
By Pooja Toshniwal PahariaAug 3 2023Reviewed by Lily Ramsey, LLM In a recent review published in Experimental & Molecular Medicine, researchers reviewed existing data on the discovery and development of gene-editing technologies and their applications in managing ocular diseases.
In contrast, advancements in non-invasive imaging and electroretinography enable real-time safety and efficacy monitoring. CRISPR-Cas9 comprises single-guide ribonucleic acid , which targets a particular sequence of DNA and Cas9, and cuts the DNA molecule at the target location, giving rise to double-stranded breakages . The endogenous machinery uses either of the two mechanisms for break repair: homology-directed repair or non-homologous end joining .
In recent years, the fourth-generation CBE and phage-assisted non-continuous evolution , and continuous evolution methods have been applied to improve the efficiency and compatibility of the original ABEs. Applications of gene editing in managing ocular diseases The eye is an ideal candidate for gene therapy and genome-editing approaches due to its small size, immune privilege status, compartmentalization, and easy accessibility.
CRISPR-Cas9-mediated gene editing can potentially target the collagen type VIII alpha two chain mutation in the early-onset Fuchs endothelial corneal dystrophy mouse model, potentially circumventing the need for transplantation.
United States Latest News, United States Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
The role of transcription factors in shaping regulatory T cell identity - Nature Reviews ImmunologyRegulatory T cells (Treg cells) are controlled by a raft of transcription factors besides Forkhead box protein 3 (FOXP3). As detailed in this Review, these accessory transcription factors act alone or together with FOXP3 to coordinate Treg cell specification and function, and account for heterogeneity of niche-specific Treg cells.
Read more »
An electrogenetic interface to program mammalian gene expression by direct current - Nature MetabolismHuang et al. develop an interface to allow electrode-mediated stimulation of gene expression in human cells, utilizing direct current-generated reactive oxygen species to stimulate transgene expression downstream of the KEAP1–NRF2 biosensor. In a type 1 diabetic mouse model, this interface is demonstrated to ameliorate hyperglycemia by stimulating insulin expression.
Read more »
Anatomical and functional maturation of the mid-gestation human enteric nervous system - Nature CommunicationsDershowitz and colleagues assess second trimester human fetal enteric nervous system development and function. They describe structural reorganization of the enteric nervous system that corresponds to gastrointestinal motility onset in ex vivo preparations.
Read more »
An eco-lodge in Uganda’s tropical forest invites guests to commune with natureAn eco-lodge by Uganda-based multidisciplinary design studio Localworks, offers rare, sustainability-minded experiences in the country's tropical forests
Read more »
SunRay Kelley wanted to build in rhythm with nature, his teacherHis beard was long, his scruffy clothes were matted with mud and straw, and he went barefoot, even in snow. At times, his form seemed more animal than human
Read more »
Synergy and oxygen adaptation for development of next-generation probiotics - NatureThe anaerobic gut bacterium Faecalibacterium prausnitzii was isolated and adapted for oxygen tolerance to develop a next-generation probiotic for the treatment of conditions such as inflammatory bowel disease and type 2 diabetes.
Read more »