Abstract:
:There has been prolonged and significant interest in manipulating the genome for a wide range of applications in biomedical research and medicine. An existing challenge in realizing this potential has been the inability to precisely edit specific DNA sequences. Past efforts to generate targeted double stranded DNA cleavage have fused DNA-targeting elements such as zinc fingers and DNA-binding proteins to endonucleases. However, these approaches are limited by both design complexity and inefficient, costineffective operation. The discovery of CRISPR/Cas9, a branch of the bacterial adaptive immune system, as a potential genomic editing tool holds the promise of facile targeted cleavage. Its novelty lies in its RNA-guided endonuclease activity, which enhances its efficiency, scalability, and ease of use. The only necessary components are a Cas9 endonuclease protein and an RNA molecule tailored to the gene of interest. This lowbarrier of adoption has facilitated a plethora of advances in just the past three years since its discovery. In this review, we will discuss the impact of CRISPR/Cas9 on biomedical research and its potential implications in medicine.
journal_name
Curr Mol Medjournal_title
Current molecular medicineauthors
Go DE,Stottmann RWdoi
10.2174/1566524016666160316150847subject
Has Abstractpub_date
2016-01-01 00:00:00pages
343-52issue
4eissn
1566-5240issn
1875-5666pii
CMM-EPUB-74425journal_volume
16pub_type
杂志文章,评审abstract::Nitric oxide has emerged as one of the most important and diverse players in physiology. This small diatomic radical stunned researchers because of its existence and unique biological properties in human physiology. Over the last two decades it was found that NO often has fickle behavior in pathophysiological mechanis...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524043359854
更新日期:2004-11-01 00:00:00
abstract::Citrate is one of the major substrates for intracellular metabolism. The extracellular level of citrate is stable in blood but varies locally, with slightly increased levels in brain and high levels in prostate. Recent metabolomics research suggests that citrate level is a potential harbinger of different pathophysiol...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524016666151123104855
更新日期:2015-01-01 00:00:00
abstract::Salmonella infections are a serious public health problem in developing countries and represent a constant concern for the food industry. The severity and the outcome of a systemic Salmonella infection depends on the "virulence" of the bacteria, on the infectious dose as well as on the genetic makeup and immunological...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524023362492
更新日期:2002-06-01 00:00:00
abstract::Liver cirrhosis, an end-result of a wide variety of the liver diseases, is a world wide health problem. Because of its unique organ system, i.e., portal blood supply, bile formation and enterohepatic circulation, drug metabolism system, and sinusoidal lining cells such as Kupffer, endothelial and stellate cells, the l...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524033479528
更新日期:2003-09-01 00:00:00
abstract::Spider venom is a large pharmacological repertoire containing many biologically active peptides, which may have a potent therapeutic implication. Here we investigated a peptide toxin, named lycosin-I, isolated from the venom of the spider Lycosa singoriensis. In contrast to most spider peptide toxins adopting inhibito...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/156652412803833643
更新日期:2012-12-01 00:00:00
abstract::Type 1 diabetes (T1D) is an organ-specific autoimmune disease resulting from the specific destruction of insulin-producing pancreatic beta-cells, culminating in a state of hypoinsulinemia and hyperglycemia. Pathogenesis of T1D comprises complex series of events from the initial sensitization of antigen-presenting cell...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652409787314471
更新日期:2009-02-01 00:00:00
abstract::Non-alcoholic fatty liver disease (NAFLD) is a clinico-pathologic spectrum of conditions ranging from simple steatosis to nonalcoholic steatohepatitis (NASH). Although simple or bland steatosis follows a relatively benign clinical course, NASH can potentially progress to cirrhosis (approximately 10 to 15 percent) and ...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652410790963321
更新日期:2010-03-01 00:00:00
abstract::The unexpected encounter between the fields of HIV and chemokines has opened new perspectives for understanding the mechanisms of AIDS pathogenesis, as well as for the development of effective therapies and vaccines. Selected chemokines act as potent natural inhibitors of HIV infection, as they bind and downmodulate c...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524023361862
更新日期:2002-12-01 00:00:00
abstract:BACKGROUND:Macrophages undergo polarization or activation in response to environmental stimuli, an essential process for proper immune response. Meanwhile, excessive activation of macrophages causes autoimmune diseases. It is therefore crucial to prevent over-activation of macrophage in order to maintain the proper imm...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524017666171106114537
更新日期:2017-12-07 00:00:00
abstract::Several species of kinetoplastid protozoa cause major human infectious diseases. Trypanosoma cruzi is responsible for the fatal Chagas disease in large parts of South America, the various species of Leishmania cause a number of different human diseases with millions of patients world-wide, and the African trypanosome ...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524043360113
更新日期:2004-09-01 00:00:00
abstract:OBJECTIVE:This study aims to investigate the effect of morphine with naloxone on intestinal peristalsis and the number of interstitial cells of Cajal (ICC) in colon tissues of rabbits. METHODS:Thirty rabbits were randomly divided into five groups (n=6, each group): saline control group (NS group), low concentration of...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524019666191023112837
更新日期:2020-01-01 00:00:00
abstract::Traditionally regarded as a genetic disease of the cardiac sarcomere, hypertrophic cardiomyopathy (HCM) is the most common inherited cardiovascular disease and a significant cause of sudden cardiac death. While the most common etiologies of this phenotypically diverse disease lie in a handful of genes encoding critica...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/156652412800620020
更新日期:2012-06-01 00:00:00
abstract::Diabetic nephropathy, which represents a major form of chronic kidney disease (CKD), is a leading cause of end-stage renal disease worldwide, and is also a risk factor for cardiovascular disease (CVD). Patients with diabetes and CKD have poorer outcomes after myocardial infarction. The underlying pathogenic mechanism ...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652408785747960
更新日期:2008-09-01 00:00:00
abstract:BACKGROUND & OBJECTIVE:Interleukin 37 (IL-37) is an important regulator of the anti-inflammatory T-cell response. In this study, we investigated its expression and function in peripheral blood mononuclear cells (PBMCs) of patients with HLA-B27 associated acute anterior uveitis (AAU) and idiopathic AAU. METHODS:15 pati...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524018666180207152941
更新日期:2018-03-09 00:00:00
abstract::Until recently it was believed that TNF-induced apoptosis is mediated exclusively by TNF-RI because TNF-RII lacks death domain. However, it has been demonstrated that TNF-RII enhances TNF-RI-mediated apoptosis. In this review, I have discussed the evidence and mechanisms by which TNF-RII regulates TNF-alpha-induced ap...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524013363780
更新日期:2001-07-01 00:00:00
abstract::By eliminating the unneeded or mutant cells, programmed cell death actively participates in a wide range of biological processes from embryonic development to homeostasis maintenance in adult. Continuing efforts have identified multiple cell death pathways, with apoptosis, necrosis and autophage the mostly studied. Re...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524015666151026100339
更新日期:2015-01-01 00:00:00
abstract::Human induced pluripotent stem cells (hiPSCs) have great potential as a robust source of progenitors for regenerative medicine. The novel technology also enables the derivation of patient-specific cells for applications to personalized medicine, such as for personal drug screening and toxicology. However, the biologic...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524011313050010
更新日期:2013-06-01 00:00:00
abstract::Most lysosomal storage diseases (LSD) exhibit neurological symptoms and there has been limited success in their treatment. Innovative treatments employing novel therapy or gene therapy may offer the prospect of improvement. Recent attempts to treat the neurological forms of LSD include neural stem cell therapy, mesenc...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524023363112
更新日期:2002-02-01 00:00:00
abstract::Endometriosis is one of the most common gynecological inflammatory diseases, occurring in adolescents and women in the reproductive age group and leading to infertility. The precise etiopathogenesis of endometriosis is unknown, but several theories concerning the phenomena involved in its development have been propose...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524015666150921105218
更新日期:2015-01-01 00:00:00
abstract::Similar to the human immunodeficiency virus (HIV), the hepatitis B virus (HBV) replicates via reverse transcription, in this case, within infected hepatocytes. Substantial advances have been achieved in the past ten years in developing and utilizing nucleoside/nucleotide analog drugs to inhibit HBV replication. Most a...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652407780059159
更新日期:2007-03-01 00:00:00
abstract::A framework was developed to provide an integrated view of mycobacterial growth and its regulation. The topics reviewed include the properties of cell cultures and their relation to properties of individual cells, cell sizes and macromolecular compositions, uptake of nutrients through the cell envelope, protein biosyn...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652407780598584
更新日期:2007-05-01 00:00:00
abstract::Although surgery and radiotherapy are highly efficient in local tumor control, distal metastases and tumor recurrence often limit therapeutic outcome. It is becoming progressively more evident that curative tumor therapy depends on the presence and maintenance of an intact immune system which has the capacity to elici...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652412803306666
更新日期:2012-11-01 00:00:00
abstract::The endoplasmic reticulum (ER) provides unique machinery for the folding and posttranslational modification of many secretory and transmembrane proteins in eukaryotic cells. The unfolded protein response (UPR) is a signal transduction network from the ER to the nucleus activated when the folding demand imposed by nasc...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652406775574569
更新日期:2006-02-01 00:00:00
abstract::Chondroitin sulfate (CS)-glycosaminoglycans (GAGs) are linear, negatively charged polysaccharides attached to CS proteoglycans that make up a major component of biological matrices throughout both central and peripheral tissues. The position of their attached sulfate groups to the CS disaccharide is predicted to influ...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524020666200915110707
更新日期:2020-09-15 00:00:00
abstract::Carcinoma-associated fibroblasts (CAFs) are activated fibroblasts during development of several cancer types, and therefore emerge as an attractive therapeutic target for cancer therapy. Drugs such as PT-100 and sibrotuzumab that block the functions of CAFs have already been in clinical trials. However, these drugs ex...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524018666171205113959
更新日期:2017-01-01 00:00:00
abstract:BACKGROUND:Liver kinase B1 (LKB1)/5'-adenosine monophosphate-activated protein kinase (AMPK) signaling, a metabolic checkpoint, plays a neuro-protective role in the pathogenesis of Alzheimer's disease (AD). Amyloid-β (Aβ) acts as a classical biomarker of AD. The aim of the present study was to explore whether berberine...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524019666190315164120
更新日期:2019-01-01 00:00:00
abstract:BACKGROUND:Seven isoforms of histone deacetylase Class III have been reported - Sirtuin (SIRT) 1-7. We recently demonstrated that EX-527, an inhibitor of SIRT1, reduces mortality in a mouse model of lethal-cecal-ligationand- puncture (CLP)-induced septic shock. Our present study was aimed at determining whether selecti...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/156652401507150903185852
更新日期:2015-01-01 00:00:00
abstract:BACKGROUND:Plasmid DNA has been widely used in vaccination as well as in cell and gene therapy. It exists in multiple isoforms, including supercoiled, nicked or open circular and linear forms. Regulatory agencies recommend having more than 80% of the supercoiled isoform for the bulk release of plasmid products; thus, i...
journal_title:Current molecular medicine
pub_type: 信件
doi:10.2174/1566524020666200427110452
更新日期:2020-04-27 00:00:00
abstract:OBJECTIVE:The study aimed to evaluate the preventive effects of hydroxycitric acid(HCA) for stone formation in the glyoxylate-induced mouse model. MATERIALS AND METHODS:Male C57BL/6J mice were divided into a control group, glyoxylate(GOX) 100 mg/kg group, a GOX+HCA 100 mg/kg group, and a GOX+HCA 200 mg/kg group. Blood...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524020666200103141116
更新日期:2020-01-01 00:00:00
abstract::Inhibition of tumor angiogenesis suppresses tumor growth and metastatic spreading in many experimental models, suggesting that anti-angiogenic drugs may be used to treat human cancer. During the past decade more than eighty molecules that showed anti-angiogenic activity in preclinical studies were tested in clinical c...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524033479410
更新日期:2003-12-01 00:00:00