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Towards Mechanism-based Treatments for Fragile X Syndrome

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

Probiotics and Prebiotics in Pediatrics

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ISBN: 9783038979500 9783038979517 Year: Pages: 258 DOI: 10.3390/books978-3-03897-951-7 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Medicine (General) --- Pediatrics
Added to DOAB on : 2019-06-26 08:44:06
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The goal of this Special Issue, “Probiotics and Prebiotics in Pediatrics”, is to focus on the importance of pediatric nutrition with probiotics and prebiotics to improve gastrointestinal health in newborn, infants, and children.Specifically, the aim is to clarify if probiotics and prebiotics can influence gut microbiota composition and host-interaction favoring human health and preventing diseases.This new information will provide health care professionals with a widespread, clear and update evidence on probiotics and prebiotics and intestinal gut microbiota in pediatric care.

Keywords

acute diarrhea --- children --- Bacillus clausii --- efficacy --- randomized controlled trials --- breast feeding --- formula feeding --- human milk oligosaccharide --- 2?-fucosyllactose --- Lacto-N-neotetraose --- microbiota --- bifidobacteria --- acute gastroenteritis --- children --- Lactobacillus reuteri --- oral rehydration solution --- probiotics --- zinc --- probiotics --- allergy --- infants --- pediatrics --- human milk oligosaccharides --- human milk --- infant formula --- necrotizing enterocolitis --- preterm infant --- preterm infant --- probiotic --- human milk --- probiotic strain --- safety --- fecal microbiota --- protein hydrolyzed formulas --- cow’s milk protein --- tolerance acquisition --- non-IgE mediated allergy --- microbiome --- intestinal microbiota --- microbial programming --- nutritional programming --- allergy --- prevention --- neonatal --- preterm --- breast milk --- oligosaccharides --- diversity --- necrotizing enterocolitis --- sepsis --- growth --- constipation --- prebiotic --- intestinal transit time --- infant --- Bifidobacterium --- Lactobacillus --- probiotics --- asthma --- Childhood Asthma Control Test --- peak expiratory flow rate --- immunoglobulin E --- “Probiotics”[Mesh] --- “Pregnancy”[Mesh] --- “Infant, Newborn”[Mesh] --- Bifidobacterium breve --- probiotics --- paediatrics --- therapeutic microbiology --- celiac disease --- iron deficiency anemia --- gluten-free diet --- inulin --- prebiotics --- iron absorption --- hepcidin --- probiotics --- microbiota --- celiac disease --- gluten free diet --- probiotics --- functional gastrointestinal disorders --- functional abdominal pain disorders --- functional constipation --- infantile colic --- infant --- colic --- lactobacilli --- n/a --- fecal microbiota --- protein hydrolyzed formulas --- cow’s milk protein --- tolerance acquisition --- non-IgE mediated allergy --- n/a

Drug Delivery Technology Development in Canada

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ISBN: 9783039280049 9783039280056 Year: Pages: 352 DOI: 10.3390/books978-3-03928-005-6 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Medicine (General) --- Therapeutics
Added to DOAB on : 2020-01-07 09:21:22
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Canada continues to have a rich history of ground-breaking research in drug delivery within academic institutions, pharmaceutical industry and the biotechnology community.

Keywords

doxorubicin --- MG63 --- Vitamin D --- DOX-Vit D --- hypoxia-induced chemoresistance --- cisplatin --- polymeric micelle --- EGFR-targeted therapy --- STAT3 --- HIF-1 --- GE11 peptide --- pharmacological Inhibitors of HIF-1 and STAT3 --- combination therapy --- pharmacokinetics --- antibodies --- radiolabeling --- biodistribution --- mouse models --- oral formulation --- amphotericin B --- fungal infections --- parasitic infections --- developing world --- drug delivery --- liposomes --- drug delivery systems --- innovation --- lipid nanoparticles --- Metaplex --- triggered drug release --- liposomes --- ultrasound --- magnetic fields --- radiation --- oral delivery --- biological barriers --- co-delivery --- throughput --- sustained delivery --- phospholipid complex --- rosmarinic acid --- bioaccessibility --- dissolution --- TNO gastrointestinal model --- gastrointestinal simulator --- phytosterols --- tocopherols --- liposomes --- canola oil deodorizer distillate --- model orange juice --- virus --- plant --- bacteriophage --- phage display --- drug discovery --- encapsulation --- drug delivery --- blood-brain barrier --- intra-arterial chemotherapy --- malignant gliomas --- primary central nervous system lymphomas --- transdermal drug delivery --- Canada --- skin --- permeation enhancers --- oral, head and neck squamous cell carcinoma --- targeted therapies --- drug delivery systems --- nanoparticles --- controlled drug delivery --- circadian clock --- chronotherapy --- precision medicine --- cationic gemini surfactant --- melphalan --- inclusion complex --- ROESY NMR spectroscopy --- 3D spheroid --- drug-resistant melanoma --- liposome --- water miscible solvents --- remote loading --- staurosporine --- cancer --- gambogic acid --- loading gradients --- mefloquine --- child friendly formulation --- blood-brain barrier (BBB) --- drug delivery --- transient modulation --- HAV6 cadherin peptide --- adenanthin --- magnetic resonance imaging (MRI) --- medulloblastoma --- drug delivery --- pharmaceutics --- drug development --- formulation and dosage form development --- translational research --- biologicals --- small molecules --- clinical trials --- pharmacokinetics --- medical devices --- route of administration --- nifedipine --- emulsion --- flavonoids --- topical formulation --- quercetin --- photostabilizers

New Advances on Zika Virus Research

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ISBN: 9783038977643 Year: Pages: 552 DOI: 10.3390/books978-3-03897-765-0 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Internal medicine --- Medicine (General)
Added to DOAB on : 2019-04-05 10:34:31
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Abstract

Zika virus (ZIKV) is a mosquito-borne member of the Flaviviridae family that historically has been associated with mild febrile illness. However, the recent outbreaks in Brazil in 2015 and its rapid spread throughout South and Central America and the Caribbean, together with its association with severe neurological disorders—including fetal microcephaly and Guillain-Barré syndrome in adults—have changed the historic perspective of ZIKV. Currently, ZIKV is considered an important public health concern that has the potential to affect millions of people worldwide. The significance of ZIKV in human health and the lack of approved vaccines and/or antiviral drugs to combat ZIKV infection have triggered a global effort to develop effective countermeasures to prevent and/or treat ZIKV infection. In this Special Issue of Viruses, we have assembled a collection of 32 research and review articles that cover the more recent advances on ZIKV molecular biology, replication and transmission, virus–host interactions, pathogenesis, epidemiology, vaccine development, antivirals, and viral diagnosis.

Keywords

Ziks virus --- silvestrol --- antiviral --- eIF4A --- hepatocytes --- flavivirus --- arbovirus --- Zika --- sexual transmission --- testis --- prostate --- Zika virus --- ZIKV --- rhesus macaques --- Non-human primates --- NHP --- infection --- natural history --- Asian-lineage --- African-lineage --- zika virus --- ZIKV–host interactions --- viral pathogenesis --- cell surface receptors --- antiviral responses --- viral counteraction --- cytopathic effects --- microcephaly --- ZIKV-associated neurologic disorders --- Zika virus --- serology --- flavivirus --- microsphere immunoassay --- validated --- optimised --- dengue virus --- ZIKV --- reporter virus --- cryptic promoter silencing --- full-length molecular clone --- subgenomic replicon --- plasmid toxicity --- Zika virus --- dengue viruses --- flavivirus --- ELISA --- indirect immunofluorescence --- plaque reduction neutralization test --- polymerase chain reaction --- cross-reactions --- Zika virus --- flavivirus --- infectious cDNA --- replication --- gene expression --- neuropathogenesis --- viral genetic variation --- host genetic variation --- flavivirus --- Zika virus --- therapy --- host-directed antivirals --- Aedes aegypti --- RNA-seq --- insecticide resistance --- Zika virus --- detoxification and immune system responses --- Zika virus --- mosquito-borne flavivirus --- emerging arbovirus --- outbreak control --- molecular diagnostics --- laboratory preparedness --- assay standardization --- external quality assessment --- EQA --- QCMD --- flavivirus --- eye --- zika virus --- blood-retinal barrier --- ocular --- innate response --- Zika virus --- pregnancy --- fetal infection --- congenital Zika syndrome --- Asian lineage --- Zika virus --- Full-length cDNA infectious clones --- Bacterial artificial chromosome --- NS2A protein --- Zika virus --- neural progenitor cells --- neurons --- Zika virus --- antivirals --- therapeutics --- research models and tools --- flavivirus --- Zika virus (ZIKV) --- reverse genetics --- infectious clone --- full-length molecular clone --- bacterial artificial chromosome --- replicon --- infectious RNA --- Zika virus --- flavivirus --- arbovirus --- sexual transmission --- host genetic variation --- immune response --- Zika virus --- flaviviruses --- vaccines --- virus like particles --- clinical trials --- ZIKV --- NS1 protein --- Zika virus --- diagnosis --- monoclonal antibodies --- ELISA --- zika virus --- placenta cells --- microglia cells --- siRNA --- TLR7/8 --- Zika --- viral evolution --- genetic variability --- Bayesian analyses --- Zika virus --- reverse genetics --- infectious cDNA --- Tet-inducible --- MR766 --- FSS13025 --- flavivirus --- ZIKV --- NS5 --- type I IFN antagonist --- point-of-care diagnostics --- isothermal nucleic acid amplification --- nucleic acid computation --- nucleic acid strand exchange --- zika virus --- mosquito --- mosquito surveillance --- multiplex nucleic acid detection --- boolean logic-processing nucleic acid probes --- Zika virus --- flavivirus --- astrocytomas --- dsRNA --- viral fitness --- antiviral --- heme-oxygenase 1 --- Zika virus --- viral replication --- Zika virus --- antiviral compounds --- neural cells --- viral replication --- flavivirus --- Zika virus --- viral persistence --- testicular cells --- testes --- Zika virus --- prM-E proteins --- viral pathogenicity --- virus attachment --- viral replication --- viral permissiveness --- viral survival --- apoptosis --- cytopathic effects --- mutagenesis --- chimeric viruses --- human brain glial cells --- Zika virus --- flavivirus --- microRNAs --- neurons --- neuroinflammation --- anti-viral immunity --- Zika virus --- dengue virus --- secondary infections --- cross-reactions --- IgA --- IgG avidity tests

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english (4)


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2019 (4)