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New treatment perspectives in autism spectrum disorders

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Book Series: Frontiers Research Topics ISSN: 16648714 ISBN: 9782889195305 Year: Pages: 161 DOI: 10.3389/978-2-88919-530-5 Language: English
Publisher: Frontiers Media SA
Subject: Pediatrics --- Medicine (General)
Added to DOAB on : 2015-12-10 11:59:06
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Developing novel and more effective treatments that improve quality of life for individuals with autism spectrum disorders is urgently needed. To date a wide range of behavioral interventions have been shown to be safe and effective for improving language and cognition and adaptive behavior in children and adolescents with ASD. However many people with ASD can receive additional benefit from targeted pharmacological interventions. One of the major drawback in setting up therapeutics intervention is the remarkable individual differences found across individuals with ASD. As a matter of fact the medications that are currently available address only symptoms associated with ASD and not the core domains of social and communication dysfunction. The pathogenesis paradigm shift of ASD towards synaptic abnormalities moved the research to pathway to disease that involve multiple systems and that are becoming the forefront of ASD treatment and are pointing toward the development of new targeted treatments. Some new therapeutics have been tested and others are being studied. In this context single gene disorders frequently associated with ASD such as Rett Syndrome, Fragile X and Tuberous Sclerosis have been of significant aid as neurobiology of these disorders is more clear and has a potential to shed light on the altered signaling in ASD. However much research is needed to further understand the basic mechanisms of disease and the relationship to idiopathic ASD. Clinical trials in children are underway with agents directed to core symptoms and to the associated disorders in the search of new therapeutics and progress are expected with possible new option for therapeutics in ASD in the upcoming future. Children and Adolescents with ASD and their families can provide important information about their experience with new treatments and this should be a priority for future research. In addition, research performed on genetic mouse models of ASD will keep on providing useful information on the molecular pathways disrupted in the disease, thus contributing to identify novel drug targets.

Immune responses to AAV vectors, from bench to bedside

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Book Series: Frontiers Research Topics ISSN: 16648714 ISBN: 9782889195008 Year: Pages: 95 DOI: 10.3389/978-2-88919-500-8 Language: English
Publisher: Frontiers Media SA
Subject: Allergy and Immunology --- Medicine (General)
Added to DOAB on : 2015-12-03 13:02:24
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The recent wave of clinical studies demonstrating long-term therapeutic efficacy highlights the enormous potential of gene therapy as an approach to the treatment of inherited disorders and cancer. While in recent years lentiviral vectors have dominated the field of ex vivo gene therapy in man, adeno-associated virus (AAV) vectors have become the platform of choice for the in vivo gene delivery, both local and systemic.Despite the achievements in the clinic however, a number of hurdles remain to be overcome in gene therapy, these include availability of scalable vector production systems, potential issues associated with insertional mutagenesis, and concerns related to immunogenicity of gene therapeutics. For AAV vectors, clinical trials showed that immunity directed against the vector could either prevent transduction of a target tissue or limit the duration of therapeutic efficacy. Initial observations in the context of a gene therapy trial for hemophilia spurred over a decade efforts by gene therapists and immunologists to understand the mechanism and identify factors that contribute to AAV’s immunogenicity, including the prevalence of B cell and T cell immunity to wild type AAV in humans and the interaction of AAV vectors with the innate and adaptive immune system. Despite a number of important contributions in particular in the more recent past, our knowledge on the immunology of gene transfer is still rudimental; this is partly due to the fact that the basic understanding of the complex balance between tolerance and immunity to an antigen, key aspect of gene transfer with AAV, keeps evolving rapidly. However, continuing work towards a better definition of the interaction of viral vectors with the immune system has led to significant advances in the knowledge of the factors influencing the outcome of gene transfer, such as the vector dose, the immune privilege of certain tissues, and the induction of tolerance to an antigen. A better understanding of the structure-function relationship of the viral capsid has boosted the development of novel immune-escape vector variants. In addition, novel immunomodulatory strategies were established to prevent or reduce anti-capsid immunity have been developed and are being tested in preclinical models and in clinical trials. Together, these advances are bringing us closer to the goal of achieving safe and sustained therapeutic gene transfer in humans. In this research topic, a collection of Original Research and Review Articles highlights critical aspects of the interaction between gene AAV vectors and the immune system, discussing how these interactions can be either detrimental or constitute an advantage, depending on the context of gene transfer, and providing tools and resources to better understand the issue of immunogenicity of AAV vectors in gene transfer.

Natural Compounds as New Cancer Treatments

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ISBN: 9783039213658 9783039213665 Year: Pages: 128 DOI: 10.3390/books978-3-03921-366-5 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Medicine (General)
Added to DOAB on : 2019-12-09 11:49:15
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Cancer remains one of the main causes of morbidity and mortality worldwide. Although many pharmacological and clinical advances have been made, there is a constant need for new molecules to improve the overall options for treatment. Natural compounds from animal, microbial, vegetal, or fungal origin represent countless sources of new compounds that can be used as anticancer drugs, provided their activity, bioavailability, and toxicity are adequate. This book aims to compile both original articles and reviews that cover the most recent advances in the use of natural compounds for cancer treatment, and provide new objectives and advice for future research in the field of biological activity of natural compounds.

Cancer Biomarkers and Targets in Digestive Organs

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ISBN: 9783039214631 9783039214648 Year: Pages: 146 DOI: 10.3390/books978-3-03921-464-8 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Medicine (General)
Added to DOAB on : 2019-12-09 11:49:15
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Identification and development of cancer biomarkers and targets have greatly accelerated progress towards precision medicine in oncology. Studies of tumor biology have not only provided insights into the mechanisms underlying carcinogenesis, but also led to discovery of molecules that have been developed into cancer biomarkers and targets. Multi-platforms for molecular characterization of tumors using next-generation genomic sequencing, immunohistochemistry, in situ hybridization, and blood-based biopsies have greatly expanded the portfolio of potential biomarkers and targets. These cancer biomarkers have been developed for diagnosis, early detection, prognosis, and prediction of treatment response. The molecular targets have been exploited for anti-cancer therapy and delivery of therapeutic agents. This Special Issue of Biomedicines focuses on recent advances in the discovery, characterization, translation, and clinical application of cancer biomarkers and targets in malignant diseases of the digestive system. The goal is to stimulate basic and translational research and clinical collaboration in this exciting field with the hope of developing strategies for prevention and early detection/diagnosis of cancer in digestive organs, and improving therapeutic and psychosocial outcomes in patients with these malignant diseases.

Keywords

colorectal cancer --- intestinal disorder --- intestinal tumors --- zebrafish --- stereotactic body radiation therapy --- immunotherapy --- biomarkers --- Asian Cancer Research Group (ACRG) --- gastric carcinoma --- molecular profiling --- precision therapy --- pembrolizumab --- predictive biomarkers --- ramucirumab --- The Cancer Genome Atlas (TCGA) --- therapeutic targets --- trastuzumab --- biliary tract carcinoma --- chemotherapy --- clinical trial --- colorectal carcinoma --- gastric carcinoma --- gastrointestinal oncology --- hepatocellular carcinoma --- immunotherapy --- pancreatic carcinoma --- targeted therapy --- Liver transplantation --- liver graft injury --- intragraft gene expression profiles --- cell adhesion molecules --- CD274 --- HFE --- hepatocellular carcinoma --- immunohistochemistry --- molecular profiling --- next-generation sequencing --- precision medicine --- predictive biomarkers --- gastrointestinal oncology --- pancreatic carcinoma --- hepatocellular carcinoma --- biliary tract carcinoma --- gastric carcinoma --- colorectal carcinoma --- stereotactic body radiation therapy --- liver transplant --- targeted therapy --- psychosocial support --- G protein–coupled receptors --- cholecystokinin --- gastrin --- gastrin-releasing peptide --- bombesin --- neurokinin --- neurotensin --- somatostatin --- circulating tumor cells --- colorectal carcinoma --- CAM invasion assay --- phenotypic mosaics --- tumor progenitor --- biomarker --- gastrointestinal malignancies --- immunotherapy --- n/a

Nutrition During Pregnancy and Lactation: Implications for Maternal and Infant Health

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ISBN: 9783039280544 9783039280551 Year: Pages: 238 DOI: 10.3390/books978-3-03928-055-1 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Nutrition and Food Sciences --- Biology --- Science (General)
Added to DOAB on : 2020-01-30 16:39:46
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Pregnancy is a viewed as a window to future health. With the birth of the developmental origins of human adult disease hypothesis, research and clinical practice has turned its attention to the influence of maternal factors such as health and lifestyle surrounding pregnancy as a means to understand and prevent the inter-generational inheritance of chronic disease susceptibility. Outcomes during pregnancy have long-lasting impacts on both women on children. Moreover, nutrition early in life can influence growth and the establishment of lifelong eating habits and behaviors. This Special Issue on “Nutrition during Pregnancy and Lactation: Implications for Maternal and Infant Health” is intended to highlight new epidemiological, mechanistic and interventional studies that investigate maternal nutrition around the pregnancy period on maternal and infant outcomes. Submissions may include original research, narrative reviews, and systematic reviews and meta-analyses.

Keywords

milk oligosaccharides --- infant formula --- 6?-sialyllactose --- cognitive development --- sialic acid --- maternal nutrition --- breast milk --- premature delivery --- milk composition --- nutritional intervention --- pregnant women --- overweight --- obesity --- total sugars --- energy --- vitamin B12 --- micronutrients --- pregnancy --- adolescents --- folate --- growth chart --- breastfeeding --- physiological body-weight loss --- thermal control --- basal maintenance expenditure --- maternal diet quality --- pregnancy --- lactation --- infancy --- growth --- body composition --- early programming --- hepatic lipogenesis --- insulin-resistant pregnancy --- metabolic flexibility --- non-alcoholic fatty liver disease --- slow digesting carbohydrates --- pregnancy --- gestational weight gain intervention --- eating behavior --- restraint --- disinhibition --- uncontrolled and emotional eating --- fetal growth --- overweight and obesity --- generalized linear models --- passive immunization --- antibodies --- lactation --- prematurity --- proteolysis --- breast milk --- full breastfeeding --- postpartum --- weight retention --- obesity --- prenatal intervention --- meal replacements --- randomized clinical trial --- lifestyle intervention --- obesity --- RDA --- micronutrients --- hippocampus --- DNA methylation --- DNA sequencing --- iron --- neurobiology --- transcriptome --- micronutrient deficiency --- neuroplasticity --- maternal obesity --- gestational weight gain --- immunological properties --- human milk --- nutrition --- health --- Pregnancy --- obesity --- diet quality --- Healthy Eating Index --- food cravings --- mindful eating --- education --- race --- food photography --- pregnancy --- energy expenditure --- energy intake --- physical activity --- metabolic rate --- fetal development

Hurdles for Phage Therapy (PT) to Become a Reality

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ISBN: 9783039213917 9783039213924 Year: Pages: 484 DOI: 10.3390/books978-3-03921-392-4 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Science (General) --- Biology --- Microbiology
Added to DOAB on : 2019-12-09 11:49:15
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Alternative treatment modes for antibiotic-resistant bacterial pathogens have become a public health priority. Bacteriophages are bacterial viruses that infect and lyse bacterial cells. Since bacteriophages are frequently bacterial host species-specific and can often also infect antibiotic-resistant bacterial cells, they could represent ideal antimicrobials for fighting the antibiotic resistance crisis. The medical use of bacteriophages has become known as phage therapy. It is widely used in Russia, where phage cocktails are sold in pharmacies as an over-the-counter drug. However, no phage product has been registered for medical purposes outside of the former Soviet Union. The current Special Issue of Viruses contains a collection of papers from opinion leaders in the field who explore hurdles to the introduction of phage therapy in western countries. The articles cover diverse topics ranging from patent to regulatory issues, the targeting of suitable bacterial infections, and the selection and characterization of safe and efficient phage cocktails. Phage resistance is discussed, and gaps in our knowledge of phage–bacterium interactions in the mammalian body are revealed, while other articles explore the use of phages in food production and processing.

Keywords

Staphylococcus aureus --- bacteriophage --- phage therapy --- vB_SauM-fRuSau02 --- Twortlikevirus --- antibiotic --- antimicrobial resistance --- magistral preparation --- compounding pharmacy --- phage therapy --- regulatory framework --- personalized medicine --- bacteriophage --- phage --- horizontal gene transfer --- co-evolution --- phage therapy --- industrial phage application --- antimicrobial resistance (AMR) --- Germany --- pH stability --- phage-host interactions --- genomics --- antibiotic-resistance --- phage preparation --- lysins --- biofilms --- typhoid fever --- Salmonella Typhi --- extended-spectrum beta lactamases (ESBL) --- Democratic Republic of the Congo --- bacteriophages --- MALDI-MS --- Staphylococcus --- bacteriophages --- phage therapy --- Kayvirus --- Viral proteins --- bacteriophage --- therapy --- phage therapy --- bacterial disease --- infection --- target selection --- Bacteriophage --- phage therapy --- resistance --- adaptation --- prophage --- production --- regulation --- phage therapy --- viral genomes --- best practices --- IND --- high-throughput sequencing --- bacteriophages --- phages --- food safety --- foodborne illness --- phage therapy --- history of science --- science communication --- bacteriophage --- phage therapy --- sustainable agriculture --- zoonosis --- antibiotic resistance --- phage therapy --- experimental therapy --- phage cocktails --- anti-phage antibodies --- prophage --- immunomodulation --- phage therapy --- evolution --- bacterial resistance --- virulence --- Listeria ivanovii --- bacteriophages --- alginate --- production --- disinfection --- phagodisinfection --- virus–host interactions --- bacteriophage efficacy --- gastrointestinal tract --- phage therapy --- bacteriophage --- phage therapy --- antimicrobial resistance --- antibiotic --- global health --- developing countries --- infectious disease --- bacteriophage --- phage --- phage therapy --- phage-resistance --- phage therapy --- bacterial infection --- capsule depolymerase --- antibiotic --- animal model --- bacterial resistance --- bacteriophage --- immunology --- innate immunity --- adaptive immunity --- human host --- phage-human host interaction --- bacterial infection --- antibiotic resistance --- bacteriophage --- antibiotic therapy --- phage therapy --- cases report --- abortive infection --- prophage --- adsorption --- Enterococcus --- rhamnopolysaccharide --- bacteriophage --- phage therapy --- Staphylococcus aureus --- biofilm --- antimicrobial --- frequency of resistance --- phage sensitivity --- resistance management --- nontraditional antibacterial --- bacteriophages --- phage therapy --- antibiotic resistance --- Pseudomonas aeruginosa --- Escherichia coli --- Staphylococcus aureus --- Brussels --- Belgium --- phage biocontrol --- patent landscape --- crop production --- bacteriophage --- phage therapy --- multidrug-resistant bacteria --- antimicrobial resistance --- bacteriophage therapy --- compassionate use --- antibiotic resistance --- phage therapy --- PTMP --- ATMP --- regulatory framework --- pharmaceutical paradigm shift --- clinical trial --- magistral formula --- personalized medicine --- phage therapy --- E. faecalis --- OrthoMCL --- antimicrobial resistance --- capsule --- Galleria mellonella --- Klebsiella pneumoniae --- phage therapy --- n/a --- antimicrobial resistance --- bacteriophage --- personalised medicines --- phage therapy --- pharmaceutical legislation --- regulatory framework

Transmucosal Absorption Enhancers in the Drug Delivery Field

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ISBN: 9783039218486 9783039218493 Year: Pages: 406 DOI: 10.3390/books978-3-03921-849-3 Language: English
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Therapeutics --- Medicine (General)
Added to DOAB on : 2020-01-30 16:39:46
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Development of strategies to assist the movement of poorly permeable molecules across biological barriers has long been the goal of drug delivery science. In the last three decades, there has been an exponential increase in advanced drug delivery systems that aim to address this issue. However, most proprietary delivery technologies that have progressed to clinical development are based on permeation enhancers (PEs) that have a history of safe use in man. This Special Issue entitled “Transmucosal Absorption Enhancers in the Drug Delivery Field” aims to present the current state-of-the-art in the application of PEs to improve drug absorption. Emphasis is placed on identification of novel permeation enhancers, mechanisms of barrier alteration, physicochemical properties of PEs that contribute to optimal enhancement action, new delivery models to assess PEs, studies assessing safety of PEs, approaches to assist translation of PEs into effective oral, nasal, ocular and vaginal dosage forms and combining PEs with other delivery strategies.

Keywords

absorption enhancers --- sugar-based surfactants --- biocompatibility studies --- transmucosal drug delivery --- intestinal permeation enhancers --- sodium cholate (NaC) --- N-dodecyl-?-D-maltoside (DDM) --- small intestine --- enterocyte --- brush border --- tryptophan --- oral delivery --- insulin --- GLP-1 --- intestinal absorption --- amino acid --- cell-penetrating peptide --- combined microsphere --- chitosan --- cyclodextrin --- nasal delivery --- nose to brain transport --- penetration enhancer --- nasal formulation --- in vivo studies --- nose to brain delivery --- antiepileptic drug --- drug delivery --- block copolymers --- thermogel system --- chitosan derivatives --- amphiphilic polymers --- polymeric micelles --- quaternization --- curcumin --- intestinal delivery --- mucoadhesiveness --- cervicovaginal tumors --- cationic functionalization --- imatinib --- nanocrystals --- in situ hydrogel --- bioenhancer --- cytochrome P450 --- drug absorption enhancer --- efflux --- metabolism --- P-glycoprotein --- pharmacokinetic interaction --- tight junction --- Aloe vera --- gel --- whole leaf --- absorption enhancement --- Caco-2 --- confocal laser scanning microscopy --- F-actin --- FITC-dextran --- tight junctions --- transepithelial electrical resistance --- permeation enhancer --- oral delivery --- formulation --- permeability --- safety --- simulated intestinal fluid --- hydrophobization --- epithelium --- compound 48/80 --- chitosan --- nanoparticles --- mast cell activator --- vaccine adjuvant --- nasal vaccination --- absorption enhancer --- antimicrobial peptide --- Caco-2 --- claudin --- cell-penetrating peptide (CPP) --- drug delivery --- intestinal epithelial cells --- KLAL --- PN159 --- tight junction modulator --- oral macromolecule delivery --- oral peptides --- sodium caprate --- salcaprozate sodium --- epithelial permeability --- epithelial transport --- nasal permeability --- nose-to-brain --- simvastatin --- nanocapsules --- mucoadhesion --- CNS disorders --- chitosan --- nasal --- pulmonary --- drug administration --- absorption enhancers --- nanoparticle --- and liposome --- absorption enhancer --- gemini surfactant --- intestinal absorption --- poorly absorbed drug --- Caco-2 cells --- PTH 1-34 --- teriparatide --- nasal delivery --- pharmacokinetics --- osteoporosis --- man --- sheep --- clinical trial --- preclinical --- Caco-2 --- intestinal absorption --- nanomedicine --- nanoparticle --- oral delivery --- transferrin --- ocular drug delivery --- cornea --- penetration enhancers --- ocular conditions --- ophthalmology --- permeation enhancers --- absorption modifying excipients --- oral delivery --- nasal delivery --- ocular delivery --- vaginal delivery --- transmucosal permeation

Human Milk and Lactation

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ISBN: 9783039289233 / 9783039289240 Year: Pages: 368 DOI: 10.3390/books978-3-03928-924-0 Language: eng
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Science (General) --- Biology --- Nutrition and Food Sciences
Added to DOAB on : 2020-06-09 16:38:57
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Human milk is uniquely tailored to meet infants’ specific nutritional requirements. However, it is more than just “milk”. This dynamic and bioactive fluid allows mother–infant signalling over lactation, guiding the infant in the developmental and physiological processes. It exerts protection and life-long biological effects, playing a crucial role in promoting healthy growth and optimal cognitive development. The latest scientific advances have provided insight into different components of human milk and their dynamic changes over time. However, the complexity of human milk composition and the synergistic mechanisms responsible for its beneficial health effects have not yet been unravelled. Filling this knowledge gap will shed light on the biology of the developing infant and will contribute to the optimization of infant feeding, particularly that of the most vulnerable infants. Greater understanding of human milk will also help in elucidating the best strategies for its storage and handling. The increasing knowledge on human milk’s bioactive compounds together with the rapidly-advancing technological achievements will greatly enhance their use as prophylactic or therapeutic agents. The current Special Issue aims to welcome original works and literature reviews further exploring the complexity of human milk composition, the mechanisms underlying the beneficial effects associated with breastfeeding, and the factors and determinants involved in lactation, including its promotion and support.

Keywords

human milk --- preterm --- term --- infants --- lipids --- fatty acids --- human milk fortification --- docosahexaenoic acid (DHA) --- mothers’ own milk --- donor human milk --- arachidonic acid (ARA) --- eicosapentaenoic acid (EPA) --- bioactive factors --- carotenoids --- dietary intake --- high-performance liquid chromatography (HPLC) --- human milk --- lactation --- maternal diet --- prospective study --- Human Milk --- Preterm infant --- Phospholipids --- Lipidomics --- Milk Fat Globule Membrane --- breastfeeding --- late preterm --- protective factors --- promotion of breastfeeding --- breastfeeding support --- mammary gland --- immune-active proteins --- proteases --- protease inhibitors --- digestive tract --- antenatal --- expressing --- pregnancy --- video instruction --- colostrum --- breastfeeding --- online --- clinical trial --- lactation --- infants --- breastfeeding --- lactating women --- human milk benefits --- colostrum --- milk therapy --- bioactive factors --- 2?-fucosyllactose (2?FL) --- 3?-sialyllactose (3?SL) --- disialyllacto-N-tetraose (DSLNT) --- human milk oligosaccharides (HMO) --- milk group --- secretor --- Lewis --- lactation --- preterm --- adipokines --- antioxidants --- breastfeeding --- cytokines --- growth factors --- human milk --- preterm --- term --- neonate --- infant --- macronutrients --- protein --- fat --- lactose --- nutrition --- passive immunization --- maternal immunoglobulins --- lactation --- prematurity --- flu vaccine --- human milk --- human milk --- omega-3 fatty acids --- docosahexaenoic acid --- eicosapentaenoic acid --- ?-linolenic acid --- dietary intake --- food frequency questionnaire --- vitamin D deficiency --- supplementation --- breastfeeding --- mothers --- infants --- human milk --- targeted metabolomics --- amino acids --- lipid metabolites --- LC-MS --- flow injection analysis --- human milk --- protein --- glycoprotein --- endogenous peptide --- breastfeeding --- hormones --- adipokines --- breast milk --- growth --- body composition --- term infant --- human milk --- term --- preterm --- calcium --- phosphorus --- magnesium --- zinc --- iron --- copper --- iodine --- selenium --- potassium --- sodium --- n-9 fatty acid --- nervonic acid --- human milk fat --- infant formula --- lactational stage --- donor human milk --- high pressure processing --- carotenoids --- antioxidant capacity --- lipids --- bile salt stimulated lipase --- preterm --- retinol --- ?-tocopherol --- inadequate intake --- nutritional status --- breast milk --- undernourishment --- dietary assessment --- multiple source method --- human milk --- celiac disease --- hormones --- fatty acids --- duration of lactation --- country --- carbohydrate --- fat --- breastfeeding difficulties --- early breastfeeding cessation --- term infants --- breastfeeding support --- human milk --- donor milk --- choline --- phosphocholine --- storage --- pasteurization --- milk banking --- pumping --- breastfeeding --- lactation --- n/a

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