Search results for " CRISPR"
showing 5 items of 5 documents
Genetic determinants of ATR inhibitor sensitivity and resistance in Gastric Cancer
2019
Synthetic lethal approaches in identifying genetic determinants of drug response is a powerful method in selecting patents for targeted cancer therapies. Ataxia-Telangiectasia Mutated (ATM) and Rad3-related protein kinase (ATR) is a valuable target to inhibit the DNA damage repair (DDR) pathway, that has been shown to be particularly effective in cancer cells harbouring other DDR defects, including truncating mutations in ARID1A, found in the 20% of gastric cancer (GC) patients. Although ATR inhibitors (ATRi) are emerging as promising cancer therapies, resistance mechanisms inevitably arise from these drugs as monotherapy, emphasising the importance of identifying genetic determinants of re…
Crispr gene editing in lipid disorders and atherosclerosis: Mechanisms and opportunities
2021
Elevated circulating concentrations of low-density lipoprotein cholesterol (LDL-C) have been conclusively demonstrated in epidemiological and intervention studies to be causally associated with the development of atherosclerotic cardiovascular disease. Enormous advances in LDL-C reduction have been achieved through the use of statins, and in recent years, through drugs targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), a key regulator of the hepatic LDL-receptor. Existing approaches to PCSK9 targeting have used monoclonal antibodies or RNA interference. Although these approaches do not require daily dosing, as statins do, repeated subcutaneous injections are nevertheless neces…
Citrus biotechnology
2020
Agricultural crops that can better withstand the changing climatic and pathogen landscape have been produced through natural selection throughout the millennia and, in recent years, through the process of human-assisted plant breeding and selection. However, a lack of genetic diversity in many commercially cultivated crops (due to monoculture) has made them more vulnerable to biotic and abiotic stresses (Esquinas-Alcazar, 2005; Keneni et al., 2012). Citrus, belonging to the Rutaceae family, is one of the most important commercial woody fruit crops in the tropical and subtropical areas of the world. The origin of citrus is traced back to parts of tropical and subtropical Southeast Asia (Wu e…
Investigating CRISPR-CAS13b as a tool for the RNA editing of CFTR mRNA with premature stop codon
2020
Background and Rationale Some CF patients are compound heterozygous or homozygous for nonsense mutations in the CFTR gene. Mutant CFTR gene coding for transcripts with premature termination codons (PTCs) is responsible for truncated CFTR protein and for a severe form of the disease. In a precision medicine framework the “REPAIRv2” (RNA Editing for Programmable A to I Replacement v2) tool, developed in the laboratory of Dr. Feng Zhang (USA), seems a good alternative to restore the full-length CFTR protein by editing its mRNA containing PTCs. This new approach is based on the possibility of targeting a deaminase enzyme (huADAR2) to a specific Adenosine, to be edited to Inosine (G analogue), o…
La rivoluzione dei semi senza sesso
2018
La tecnica di editing genetico CRISPR segna un altro importantissimo successo