Search results: Found 4

Listing 1 - 4 of 4
Sort by
Building Strategies for Porcine Cancer Models

Authors: --- --- ---
Book Series: Frontiers Research Topics ISSN: 16648714 ISBN: 9782889456505 Year: Pages: 75 DOI: 10.3389/978-2-88945-650-5 Language: English
Publisher: Frontiers Media SA
Subject: Science (General) --- Genetics
Added to DOAB on : 2019-01-23 14:53:43
License:

Loading...
Export citation

Choose an application

Abstract

The eBook "Building Strategies for Porcine Cancer Models" presents a series of articles demonstrating the state-of-the-art developments in pig models for cancer research. Renowned researchers dedicated to the reproduction, genomic and biological engineering of the pig model for biomedicine contribute to this special research area. Although advances in these areas are occurring at surprising speeds, they are still far from realizing all the potential benefits that this biological model could provide to science. The current biomedical models may limit the frontier of knowledge in the cancer research.

Gene Silencing and Editing Strategies for Neurodegenerative Diseases

Authors: ---
Book Series: Frontiers Research Topics ISSN: 16648714 ISBN: 9782889455515 Year: Pages: 115 DOI: 10.3389/978-2-88945-551-5 Language: English
Publisher: Frontiers Media SA
Subject: Science (General) --- Neurology
Added to DOAB on : 2019-01-23 14:53:42
License:

Loading...
Export citation

Choose an application

Abstract

Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation and deposition of misfolded proteins, 2) common molecular mechanisms leading to neurodegeneration, and 3) certain overlap in symptoms and clinical features. To date, there is no cure that could stop or delay the progression of these diseases. The advent of advanced gene therapy techniques such as gene silencing and gene editing opened a new avenue for the development of therapeutic strategies for NDs.The discovery of the RNA interference (RNAi) mechanism, in 1998, by Andrew Fire and Craig Mello allowed an important boost to the gene therapy field, providing a potential therapeutic strategy to treat inherited dominant genetic disorders. The use of small RNA sequences to control the expression of disease-causing genes rapidly implemented in the preclinical studies for different diseases. In the field of NDs, several successful studies using this technology proved its potential as a therapeutic option. However, issues like the type of delivery system (non-viral versus viral) or the potential toxicity of the small RNA molecules, made the translation of gene silencing therapeutics to human application very slow and difficult.Recently, a new hope in the gene therapy field emerged with the development of gene editing techniques like TALENs or CRISPR/Cas9 systems. The opportunity of editing or deleting gene sequences drove the scientific community euphoric, with an enormous increase in the number of published studies using this type of techniques. Recently, the first clinical trial using one of these systems was approved in China. For NDs, gene-editing technology also represents an important therapeutic option, and the first preclinical studies are now being published, showing the potential accomplishment for this technology.

Towards Mechanism-based Treatments for Fragile X Syndrome

Authors: ---
ISBN: 9783039215058 / 9783039215065 Year: Pages: 250 DOI: 10.3390/books978-3-03921-506-5 Language: eng
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Science (General) --- Biology
Added to DOAB on : 2019-12-09 11:49:15
License:

Loading...
Export citation

Choose an application

Abstract

It has been more than 25 years since the identification of the FMR1 gene and the demonstration of the causative role of CGG-repeat expansion in the disease pathology of fragile X syndrome (FXS), but the underlying mechanisms involved in the expansion mutation and the resulting gene silencing still remain elusive. Our understanding of the pathways impacted by the loss of FMRP function has grown tremendously, and has opened new avenues for targeted treatments for FXS. However, the failure of recent clinical trials that were based on successful preclinical studies using the Fmr1 knockout mouse model has forced the scientific community to revisit clinical trial design and identify objective outcome measures. There has also been a renewed interest in restoring FMR1 gene expression as a possible treatment approach for FXS. This special issue of Brain Sciences highlights the progress that has been made towards understanding the disease mechanisms and how this has informed the development of treatment strategies that are being explored for FXS.

Keywords

fragile X syndrome --- clinical trials --- targeted treatments --- drug development --- fragile X syndrome --- clinical trials --- treatment development --- best practices --- fragile X syndrome --- newborn screening --- early identification --- fragile X syndrome --- X chromosome --- females --- FMR1 --- anxiety --- avoidance --- cognition --- behavior --- brain --- Fragile X --- FMRP --- Fxr2 --- Fmr1 --- fragile X syndrome --- executive function --- working memory --- set-shifting --- cognitive flexibility --- inhibitory control --- attention --- planning --- processing speed --- Fragile X syndrome 1 --- Fragile X-associated Tremor/Ataxia Syndrome 2 --- CRISPR 3 --- Trinucleotide Repeat 4 --- Gene editing --- fragile X syndrome --- FMR1 gene --- voice of the person --- voice of the patient --- characteristics that have the greatest impact --- developmental disorders --- fragile X syndrome --- language development --- automated vocal analysis --- adeno-associated virus --- autism spectrum disorders --- cerebral spinal fluid --- fragile X mental retardation protein --- neurodevelopmental disorders --- viral vector --- fragile X syndrome --- gene reactivation --- RNA:DNA hybrid --- FMRP --- histone methylation --- DNA methylation --- FMR1 --- PRC2 --- fragile X syndrome --- unstable repeat diseases --- epigenetic gene silencing --- DNA methylation --- repeat instability --- pluripotent stem cells --- CGG Repeat Expansion Disease --- DNA instability --- expansion --- contraction --- mismatch repair (MMR) --- base excision repair (BER) --- transcription coupled repair (TCR) --- double-strand break repair (DSBR) --- Non-homologous end-joining (NHEJ) --- mosaicism --- protein synthesis --- Fragile X Syndrome --- biomarker --- iPSC --- fibroblast --- lymphoblast --- fragile X syndrome --- molecular biomarkers --- FMR1 --- FMRP --- intellectual disability --- Fmr1 KO mouse --- ASD --- n/a

Molecular Research of Endometrial Pathophysiology

Authors: ---
ISBN: 9783039214952 / 9783039214969 Year: Pages: 378 DOI: 10.3390/books978-3-03921-496-9 Language: eng
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Social Sciences --- Sociology
Added to DOAB on : 2019-12-09 16:10:12
License:

Loading...
Export citation

Choose an application

Abstract

The endometrium has been the subject of intense research in a variety of clinical settings, because of its importance in the reproductive process and its role in women’s health. In the past 15 years, significant efforts have been invested in defining the molecular phenotype of the receptive phase endometrium as well as of various endometrial pathologies. Although this has generated a wealth of information on the molecular landscape of human endometrium, there is a need to complement this information in light of the novel methodologies and innovative technical approaches. The focus of this International Journal of Molecular Sciences Special Issue is on molecular and cellular mechanisms of endometrium and endometrium-related disorders. The progress made in the molecular actions of steroids, in the metabolism of steroids and intracrinology, in endometrial intracellular pathways, in stem cells biology, as well as in the molecular alterations underlying endometrium-related pathologies has been the focus of the reviews and papers included.

Keywords

RANK --- endometrium --- endometrial cancer --- prognosis --- immunohistochemistry --- gene expression --- endometriosis --- developmental pathway --- pathogenomics --- mesenchymal stem cells --- endometrial cancer --- mtDNA mutations --- deficit of complex I --- antioxidant response --- mitochondrial biogenesis --- mitochondrial dynamics --- mitophagy --- miRNA --- lncRNAs --- endometrial cancer --- endometriosis --- chronic endometritis --- cell contacts --- tight junction --- adherens junction --- gap junction --- endometrium --- implantation --- decidualization --- endometriosis --- endometrial cancer --- liquid biopsy --- uterine aspirate --- circulating tumour cells (CTCs) --- circulating tumour DNA (ctDNA) --- exosomes --- Vitamin D --- endometrium --- endometrial cancer --- endometrial cancer --- preclinical models --- translational research --- endometrial cancer --- type II endometrial carcinoma --- targeted therapy --- kinase inhibitor --- molecular marker --- protein kinase --- protein phosphatase --- PP2A --- PPP2R1A --- SMAP --- endometriosis --- infertility --- niche --- inflammation --- immunomodulation --- mesenchymal stem cell --- orthoxenograft --- uterine cancer --- avatar --- murine models --- personalized medicine --- targeted therapy --- preclinical studies --- translational research --- endometriosis --- TRP channels --- endometrial stromal cells --- eutopic and ectopic endometrium --- endometrial cell --- pathway --- proliferation --- decidualization --- migration --- angiogenesis --- regeneration --- breakdown --- implantation --- endometrial cancer --- orthotopic xenograft model --- estrogen dependent --- bioluminescence imaging --- contrast-enhanced CT scan --- endometrium --- adult stem cells --- endometrial regeneration --- stem cell markers --- endometriosis --- endometrial cancer --- decidualisation --- oestradiol --- aromatase --- testosterone --- dehydroepiandrosterone (DHEA) --- endometriosis --- endometrial cancer --- sulfatase --- endometriosis --- ectopic stroma --- microRNA --- small RNA sequencing --- EDN1 --- HOXA10 --- miR-139-5p --- miR-375 --- CTCF --- tumour suppressor gene --- haploinsufficiency --- zinc finger --- CRISPR/Cas9 --- cancer --- endometrial cancer --- gene editing --- phosphoinositide 3-kinase --- PIK3CA --- PIK3CB --- p110? --- p110? --- endometrial cancer --- LGR5 --- endometrium --- endometriosis --- menstrual cycle --- macrophages

Listing 1 - 4 of 4
Sort by
Narrow your search