• protein
  • They mapped the kinase interactions across the entire genome, and discovered that ERK2, a protein that belongs to the ERK signaling family, targets important sites such as non-coding genes and histones, cell cycle, metabolism and also stem cell-specific genes. (nanowerk.com)
  • This critical role of Stat3 in the molecular pathogenesis of many tumors provides a validation for targeting this protein for cancer drug discovery [reviewed in Refs. (aacrjournals.org)
  • These viruses, when modified by changing their coat protein, can infect axolotl cells only when they have been experimentally manipulated to express the receptor for that coat protein, thus allowing for the possibility of targeting specific cell types. (biologists.org)
  • However, the form of enzyme present in the circulation does not correspond to the protein as encoded by the gene: the main fraction of the active enzyme in the circulation has a molecular weight of 730 kD and is probably bound in a complex to other proteins. (wikipedia.org)
  • Cluster of differentiation 97 is a protein also known as BL-Ac[Fencoded by the ADGRE5 gene. (wikipedia.org)
  • With its sequence interrupted, the altered gene in most cases will be translated into a nonfunctional protein, if it is translated at all. (wikipedia.org)
  • Entrectinib is a pan-NTRK as well as an ALK and ROS1 tyrosine kinase inhibitor has been found useful in treating a single patient with ETV6-NRTK3 fusion gene-associated mammary analogue secretory carcinoma and has lend support to the clinical development of NTRK3-directed tyrosine kinase inhibitors to treat ETV6-NTRK3 fusion protein associated malignancies. (wikipedia.org)
  • Targeting with antibody generated to the peptide representing the first nine amino acids of Mig-7 detects cells expressing this protein and significantly inhibits carcinoma cell invasion in vitro. (wikipedia.org)
  • Targeting with short hairpin RNA, specific to decrease Mig-7 protein levels, inhibits carcinoma cell invasion and metastasis as well as returns ERK1/2, Akt, GSK, and S6 kinase to normal phosphorylation states through reactivation of PP2A. (wikipedia.org)
  • Generally, efforts focused on administering a gene that causes a needed protein to be expressed. (wikipedia.org)
  • Ubiquitin-protein ligase E3A (UBE3A) also known as E6AP ubiquitin-protein ligase (E6AP) is an enzyme that in humans is encoded by the UBE3A gene. (wikipedia.org)
  • Like mutations within the gene, these chromosomal changes prevent any functional ubiquitin protein ligase E3A from being produced in the brain. (wikipedia.org)
  • mutations
  • The fact that kinase-activating mutations in the c-Met gene have not been reported in medulloblastomas indicates that HGF:c-Met signaling in these tumors is driven by a ligand-dependent, autostimulatory loop, which could be interrupted via the high specificity and avidity of monoclonal antibodies (mAbs). (aacrjournals.org)
  • The method can be used to delete a gene, remove exons, add a gene, and introduce point mutations. (wikipedia.org)
  • Daughter cells that inherit these wrong bases carry mutations from which the original DNA sequence is unrecoverable (except in the rare case of a back mutation, for example, through gene conversion). (wikipedia.org)
  • Originally, naturally occurring mutations were identified and then gene loss or inactivation had to be established by DNA sequencing or other methods. (wikipedia.org)
  • Mutations within the UBE3A gene are responsible for some cases of Angelman syndrome and Prader-Willi syndrome. (wikipedia.org)
  • vivo
  • In this review, we provide a brief summary of different approaches developed for dissecting tissue clock function in the metabolic context in rodents, compare their strengths and weaknesses, and suggest new strategies in assessing tissue clock output and the consequences of circadian clock disruption in vivo . (frontiersin.org)
  • Altogen Labs offers preclinical services including: development of stable cell lines, in vivo biodistribution studies, and RNAi gene silencing services. (wikipedia.org)
  • In addition, other in vivo services including biomarker analysis, immune response studies, pharmacokinetics, pharmacodynamics, and siRNA delivery and tissue targeting are available. (wikipedia.org)
  • Targeting of Mig-7 with Mig-7-specific peptides significantly stimulates breast cancer patients' immune cells' killing of breast carcinoma cells ex vivo. (wikipedia.org)
  • retinal
  • Retinal gene therapy holds great promise in treating different forms of non-inherited and inherited blindness. (wikipedia.org)
  • These results were widely seen as a success in the gene therapy field, and have generated excitement and momentum for AAV-mediated applications in retinal disease. (wikipedia.org)
  • In retinal gene therapy, the most widely used vectors for ocular gene delivery are based on adeno-associated virus. (wikipedia.org)
  • sequences
  • Placing Lox sequences appropriately allows genes to be activated, repressed, or exchanged for other genes. (wikipedia.org)
  • Without methods to account for this "context dependence", the standard modular assembly procedure often fails unless it is used to recognize sequences of the form (GNN)N. Numerous selection methods have been used to generate zinc-finger arrays capable of targeting desired sequences. (wikipedia.org)
  • The construct is engineered to recombine with the target gene, which is accomplished by incorporating sequences from the gene itself into the construct. (wikipedia.org)
  • This is done by introducing short sequences called loxP sites around the gene. (wikipedia.org)
  • This germ-line can then be crossed to another germline containing Cre-recombinase which is a viral enzyme that can recognize these sequences, recombines them and deletes the gene flanked by these sites. (wikipedia.org)
  • therapeutic
  • Two mutant CDK4 isoforms (R24C, R24L) similarly stimulated T cell responses in vitro and were analyzed as therapeutic targets for TCR gene therapy. (jci.org)
  • The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. (wikipedia.org)
  • Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effects. (wikipedia.org)
  • mice
  • In this study, we analyzed whether tissue ACE deficiency in ACE-knockout mice type-2 would affect their oxidative status. (ahajournals.org)
  • Moreover, by crossbreeding the ACE-knockout mice with atherosclerotic apolipoprotein E (apo E)-deficient (E 0 ) mice, we questioned whether tissue ACE deficiency affects atherogenesis. (ahajournals.org)
  • Finally, 2 animals genotyped as homozygous-knockout for both ACE and APOE genes (ACE − / − /E 0 ), exhibited a striking reduction of 86% in their atherosclerotic lesion area compared with E 0 mice. (ahajournals.org)
  • knockout
  • Conclusions- Reduction of tissue ACE with the ACE-knockout mouse type-2 model inhibited oxidative stress and atherogenesis. (ahajournals.org)
  • Use of ACE inhibitors or knockout of the ACE gene is supposed to lower Ang II levels and reduce Ang II proatherogenic properties. (ahajournals.org)
  • However, KO can also refer to the gene that is knocked out or the organism that carries the gene knockout. (wikipedia.org)
  • Knockout organisms or simply knockouts are used to study gene function, usually by investigating the effect of gene loss. (wikipedia.org)
  • Knocking out two genes simultaneously in an organism is known as a double knockout (DKO). (wikipedia.org)
  • Similarly the terms triple knockout (TKO) and quadruple knockouts (QKO) are used to describe three or four knocked out genes, respectively. (wikipedia.org)
  • Gene knockin is similar to gene knockout, but it replaces a gene with another instead of deleting it. (wikipedia.org)
  • tumor
  • While this constitutes only 0.000165% of the human genome's approximately 6 billion bases (3 billion base pairs), unrepaired lesions in critical genes (such as tumor suppressor genes) can impede a cell's ability to carry out its function and appreciably increase the likelihood of tumor formation and contribute to tumour heterogeneity. (wikipedia.org)
  • peripheral
  • In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia. (wikipedia.org)
  • significantly
  • 2) Functional genomics and mechanistic studies of a number of additional genes at loci significantly associated with lipid and metabolic traits, CAD, or other cardiovascular traits. (upenn.edu)
  • The frequency of gene targeting can be significantly enhanced through the use of engineered endonucleases such as zinc finger nucleases, engineered homing endonucleases, and nucleases based on engineered TAL effectors. (wikipedia.org)
  • regulates
  • In sum, these studies illustrate that the SCN regulates metabolic hormones and tissue physiology either directly or via its control on food intake rhythms. (frontiersin.org)
  • enzyme
  • Heparan sulfate 2-O-sulfotransferase 1 is an enzyme that in humans is encoded by the HS2ST1 gene. (wikipedia.org)
  • Serine protease hepsin is an enzyme that in humans is encoded by the HPN gene. (wikipedia.org)
  • Because the copy of the gene inherited from a person's father (the paternal copy) is normally inactive in the brain, a mutation in the remaining maternal copy prevents any of the enzyme from being produced in the brain. (wikipedia.org)
  • cardiac tissue
  • Engineers have developed a 'heart patch' that is just as strong and electrically active as healthy adult cardiac tissue and large enough to cover the damage caused by most heart attacks. (transplantology.org)
  • human
  • Both HGF and c-Met are frequently expressed in human medulloblastomas, and the elevated mRNA levels of these genes predict an unfavorable prognosis for patients ( 19 ). (aacrjournals.org)
  • Selected genes will then be targeted as in Aim 1 to improve human beta-cell function, as assessed by glucose responsiveness. (upenn.edu)
  • Previously used to engineer rat cell models, advances in gene targeting technologies are enabling the creation of a new wave of isogenic human disease models. (wikipedia.org)
  • The genes of the two human isoenzymes were localized in the mid-1970s. (wikipedia.org)
  • The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. (wikipedia.org)
  • Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. (wikipedia.org)
  • The UBE3A gene lies within the human chromosomal region 15q11-13. (wikipedia.org)
  • vector
  • Gene targeting requires the creation of a specific vector for each gene of interest. (wikipedia.org)
  • The vector incorporates genes into chromosomes. (wikipedia.org)
  • In all three studies, an AAV vector was used to deliver a functional copy of the RPE65 gene, which restored vision in children suffering from LCA. (wikipedia.org)
  • In these studies, an AAV vector encoding the RPE65 gene was delivered via a "subretinal injection", where a small amount of fluid is injected underneath the retina in a short surgical procedure. (wikipedia.org)
  • Cell
  • Furthermore, targeting specific cell types will be necessary to understand how regeneration of the diverse tissues within the limb is accomplished. (biologists.org)
  • Independently, Joe Z. Tsien has pioneered the use of Cre-loxP system for cell type- and region-specific gene manipulation in the adult brain where hundreds of distinct neuron types may exist and nearly all neurons in the adult brain are known to be post-mitotic. (wikipedia.org)
  • 1 Molecular Cell Biology and Gene Therapy, Max-Delbrück-Center for Molecular Medicine, Berlin, Germany. (jci.org)
  • In Aim 1, we will target the genes altered in patients with marked beta-cell hyperplasia, such as those suffering from Beckwith-Wiedemann Syndrome or Multiple Endocrine Neoplasia. (upenn.edu)
  • This technology has enabled the study of gene function in specific cell types and at specific times among living animals. (wikipedia.org)
  • widely
  • After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990, when Ashi DeSilva was treated for ADA-SCID. (wikipedia.org)
  • initially
  • The former was first found in fetal tissue, the second was found to be more abundant in adult tissue, and initially they were termed fetal and adult thymidine kinase. (wikipedia.org)
  • Many genes that were initially shown to influence life span have turned out to be involved in DNA damage repair and protection. (wikipedia.org)
  • thereby
  • This novel concept of inhibiting a microRNA in a non-targeted manner, but taking advantage of its restricted tissue distribution and thereby selectively upregulating its target genes in that tissue, may have far reaching implications for microRNA biology and tissue-specific gene targeting in general," Shalev said. (eurekalert.org)
  • chromosome 15
  • The UBE3A gene is located on the long (q) arm of chromosome 15 between positions 11 and 13, from base pair 23,133,488 to base pair 23,235,220. (wikipedia.org)
  • humans
  • The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980. (wikipedia.org)