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The efficient delivery of cellular constituents to their proper location is of fundamental importance for all cells and is of particular interest to neuroscientists, because of the unique functions and complex architecture of neurons. Protein Trafficking in Neurons examines mechanisms of protein trafficking and the role of trafficking in neuronal functioning from development to plasticity to disease. The book is divided into seven sections that review mechanisms of protein transport, the role of protein trafficking in synapse formation, exo- and endocytosis, transport of receptors, tra
Neurons. --- Neurons - metabolism. --- Physiological transport. --- Protein Transport. --- Proteins. --- Proteins - Physiological transport. --- Proteins--Physiological transport, Neurons. Protein Transport. Neurons--metabolism. . --- Proteins --- Neurons --- Protein Transport --- Metabolism --- Cells --- Metabolic Phenomena --- Nervous System --- Biological Transport --- Phenomena and Processes --- Anatomy --- Animal Biochemistry --- Human Anatomy & Physiology --- Health & Biological Sciences --- Physiological transport --- Anatomies --- Biologic Transport --- Transport, Biological --- Transport, Biologic --- Nervous Systems --- System, Nervous --- Systems, Nervous --- Metabolic Phenomenon --- Metabolic Process --- Metabolism Concepts --- Metabolism Phenomena --- Process, Metabolic --- Processes, Metabolic --- Anabolism --- Catabolism --- Metabolic Concepts --- Metabolic Processes --- Concept, Metabolic --- Concept, Metabolism --- Concepts, Metabolic --- Concepts, Metabolism --- Metabolic Concept --- Metabolism Concept --- Phenomena, Metabolic --- Phenomena, Metabolism --- Phenomenon, Metabolic --- Cell --- Protein Localization Processes, Cellular --- Protein Sorting --- Protein Targeting --- Protein Trafficking --- Gated Protein Transport --- Protein Translocation --- Transmembrane Protein Transport --- Vesicular Protein Transport --- Protein Sortings --- Protein Traffickings --- Protein Transport, Gated --- Protein Transport, Transmembrane --- Protein Transport, Vesicular --- Protein Transports --- Targeting, Protein --- Trafficking, Protein --- Traffickings, Protein --- Transmembrane Protein Transports --- Nerve Cells --- Cell, Nerve --- Cells, Nerve --- Nerve Cell --- Neuron --- Nerve cells --- Neurocytes --- Nervous system
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Following the implementation of next-generation sequencing technologies (e.g., exome and genome sequencing) in molecular diagnostics, the majority of genetic defects underlying inherited retinal disease (IRD) can readily be identified. In parallel, opportunities to counteract the molecular consequences of these defects are rapidly emerging, providing hope for personalized medicine. ‘Classical’ gene augmentation therapy has been under study for several genetic subtypes of IRD and can be considered a safe and sometimes effective therapeutic strategy. The recent market approval of the first retinal gene augmentation therapy product (LuxturnaTM, for individuals with bi-allelic RPE65 mutations) by the FDA has not only demonstrated the potential of this specific approach, but also opened avenues for the development of other strategies. However, every gene—or even every mutation—may need a tailor-made therapeutic approach, in order to obtain the most efficacious strategy with minimal risks associated. In addition to gene augmentation therapy, other subtypes of molecular therapy are currently being designed and/or implemented, including splice modulation, DNA or RNA editing, optogenetics and pharmacological modulation. In addition, the development of proper delivery vectors has gained strong attention, and should not be overlooked when designing and testing a novel therapeutic approach. In this Special Issue, we aim to describe the current state of the art of molecular therapeutics for IRD, and discuss existing and novel therapeutic strategies, from idea to implementation, and from bench to bedside.
Research & information: general --- Biology, life sciences --- induced pluripotent stem cell (iPSC) --- clustered regularly interspaced short palindromic repeats (CRISPR) --- homology-directed repair (HDR) --- Enhanced S-Cone Syndrome (ESCS) --- NR2E3 --- AAV --- retina --- gene therapy --- dual AAV --- gold nanoparticles --- DNA-wrapped gold nanoparticles --- ARPE-19 cells --- retinal pigment epithelium --- clathrin-coated vesicles --- endosomal trafficking --- retinitis pigmentosa --- autosomal dominant --- G56R --- putative dominant negative effect --- gapmer antisense oligonucleotides --- allele-specific knockdown --- Leber congenital amaurosis and allied retinal ciliopathies --- CEP290 --- Flanders founder c.4723A > --- T nonsense mutation --- Cilia elongation --- spontaneous nonsense correction --- AON-mediated exon skipping --- microRNA --- photoreceptors --- rods --- cones --- bipolar cells --- Müller glia --- retinal inherited disorders --- retinal degeneration --- antisense oligonucleotides --- Stargardt disease --- inherited retinal diseases --- splicing modulation --- RNA therapy --- ABCA4 --- iPSC-derived photoreceptor precursor cells --- cyclic GMP --- apoptosis --- necrosis --- drug delivery systems --- translational medicine --- Usher syndrome --- Leber congenital amaurosis --- RPE65 --- nonprofit --- patient registry --- translational --- protein trafficking --- protein folding --- protein degradation --- chaperones --- chaperonins --- heat shock response --- unfolded protein response --- autophagy --- therapy --- IRD --- DNA therapies --- RNA therapies --- compound therapies --- clinical trials --- Retinitis Pigmentosa GTPase Regulator --- adeno-associated viral --- Retinitis Pigmentosa (RP) --- choroideremia --- REP1 --- inherited retinal disease --- treatment --- apical polarity --- crumbs complex --- fetal retina --- PAR complex --- retinal organoids --- retinogenesis --- gene augmentation --- adeno-associated virus (AAV) --- induced pluripotent stem cell (iPSC) --- clustered regularly interspaced short palindromic repeats (CRISPR) --- homology-directed repair (HDR) --- Enhanced S-Cone Syndrome (ESCS) --- NR2E3 --- AAV --- retina --- gene therapy --- dual AAV --- gold nanoparticles --- DNA-wrapped gold nanoparticles --- ARPE-19 cells --- retinal pigment epithelium --- clathrin-coated vesicles --- endosomal trafficking --- retinitis pigmentosa --- autosomal dominant --- G56R --- putative dominant negative effect --- gapmer antisense oligonucleotides --- allele-specific knockdown --- Leber congenital amaurosis and allied retinal ciliopathies --- CEP290 --- Flanders founder c.4723A > --- T nonsense mutation --- Cilia elongation --- spontaneous nonsense correction --- AON-mediated exon skipping --- microRNA --- photoreceptors --- rods --- cones --- bipolar cells --- Müller glia --- retinal inherited disorders --- retinal degeneration --- antisense oligonucleotides --- Stargardt disease --- inherited retinal diseases --- splicing modulation --- RNA therapy --- ABCA4 --- iPSC-derived photoreceptor precursor cells --- cyclic GMP --- apoptosis --- necrosis --- drug delivery systems --- translational medicine --- Usher syndrome --- Leber congenital amaurosis --- RPE65 --- nonprofit --- patient registry --- translational --- protein trafficking --- protein folding --- protein degradation --- chaperones --- chaperonins --- heat shock response --- unfolded protein response --- autophagy --- therapy --- IRD --- DNA therapies --- RNA therapies --- compound therapies --- clinical trials --- Retinitis Pigmentosa GTPase Regulator --- adeno-associated viral --- Retinitis Pigmentosa (RP) --- choroideremia --- REP1 --- inherited retinal disease --- treatment --- apical polarity --- crumbs complex --- fetal retina --- PAR complex --- retinal organoids --- retinogenesis --- gene augmentation --- adeno-associated virus (AAV)
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In the last few years, significant breakthroughs in transcription research expanded our appreciation for the complexity of molecular controls on gene expression in mammalian cells. In Transcription Factors: Methods and Protocols, experts in the field describe state-of-the-art approaches that investigators can use to probe critical mechanisms underlying transcription factor nuclear-cytoplasmic trafficking as well as to assess the functional impact of post-translational modifications on transcription factor function. The chapters are written by prominent scientists, many of whom developed these methods, and highlight protocols that focus on specific transcription factor family members with particular relevance to human disease. Composed in the highly successful Methods in Molecular Biology™ series format, each chapter contains a brief introduction, step-by-step methods, a list of necessary materials, and a Notes section which shares tips on troubleshooting and avoiding known pitfalls. Comprehensive and current, Transcription Factors: Methods and Protocols compiles the latest techniques for elucidating controls on transcription factor intracellular localization and activity, and consequently is unlike any other methods-based text on transcriptional regulation today. .
Transcription factors --- Transcription Factors --- Research --- Methodology --- Proteins --- Investigative Techniques --- Publication Formats --- Biological Transport --- Analytical, Diagnostic and Therapeutic Techniques and Equipment --- Publication Characteristics --- Amino Acids, Peptides, and Proteins --- Metabolism --- Cytological Techniques --- Clinical Laboratory Techniques --- Laboratory Manuals --- Protein Transport --- Chemicals and Drugs --- Metabolic Phenomena --- Phenomena and Processes --- Human Anatomy & Physiology --- Health & Biological Sciences --- Animal Biochemistry --- Metabolic Phenomenon --- Metabolic Process --- Metabolism Concepts --- Metabolism Phenomena --- Process, Metabolic --- Processes, Metabolic --- Anabolism --- Catabolism --- Metabolic Concepts --- Metabolic Processes --- Concept, Metabolic --- Concept, Metabolism --- Concepts, Metabolic --- Concepts, Metabolism --- Metabolic Concept --- Metabolism Concept --- Phenomena, Metabolic --- Phenomena, Metabolism --- Phenomenon, Metabolic --- Protein Localization Processes, Cellular --- Protein Sorting --- Protein Targeting --- Protein Trafficking --- Gated Protein Transport --- Protein Translocation --- Transmembrane Protein Transport --- Vesicular Protein Transport --- Protein Sortings --- Protein Traffickings --- Protein Transport, Gated --- Protein Transport, Transmembrane --- Protein Transport, Vesicular --- Protein Transports --- Targeting, Protein --- Trafficking, Protein --- Traffickings, Protein --- Transmembrane Protein Transports --- Diagnoses and Laboratory Examinations --- Diagnosis, Laboratory --- Laboratory Diagnosis --- Laboratory Examinations and Diagnoses --- Laboratory Techniques, Clinical --- Clinical Laboratory Technique --- Diagnoses, Laboratory --- Laboratory Diagnoses --- Laboratory Technique, Clinical --- Technique, Clinical Laboratory --- Techniques, Clinical Laboratory --- Transcription Factor --- Factor, Transcription --- Factors, Transcription --- Cytologic Technics --- Cytological Technic --- Cytological Technics --- Cytological Technique --- Technic, Cytological --- Technics, Cytological --- Technique, Cytological --- Techniques, Cytological --- Cytologic Technic --- Technic, Cytologic --- Technics, Cytologic --- Cell Biology --- Biologic Transport --- Transport, Biological --- Transport, Biologic --- Investigative Technics --- Investigative Technic --- Investigative Technique --- Technic, Investigative --- Technics, Investigative --- Technique, Investigative --- Techniques, Investigative --- Gene Products, Protein --- Gene Proteins --- Protein Gene Products --- Proteins, Gene --- Genetic transcription factors --- methods --- Cytology. --- Cell Biology. --- Cell biology --- Cellular biology --- Biology --- Cells --- Cytologists --- Transcription factors - Research - Methodology
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Following the implementation of next-generation sequencing technologies (e.g., exome and genome sequencing) in molecular diagnostics, the majority of genetic defects underlying inherited retinal disease (IRD) can readily be identified. In parallel, opportunities to counteract the molecular consequences of these defects are rapidly emerging, providing hope for personalized medicine. ‘Classical’ gene augmentation therapy has been under study for several genetic subtypes of IRD and can be considered a safe and sometimes effective therapeutic strategy. The recent market approval of the first retinal gene augmentation therapy product (LuxturnaTM, for individuals with bi-allelic RPE65 mutations) by the FDA has not only demonstrated the potential of this specific approach, but also opened avenues for the development of other strategies. However, every gene—or even every mutation—may need a tailor-made therapeutic approach, in order to obtain the most efficacious strategy with minimal risks associated. In addition to gene augmentation therapy, other subtypes of molecular therapy are currently being designed and/or implemented, including splice modulation, DNA or RNA editing, optogenetics and pharmacological modulation. In addition, the development of proper delivery vectors has gained strong attention, and should not be overlooked when designing and testing a novel therapeutic approach. In this Special Issue, we aim to describe the current state of the art of molecular therapeutics for IRD, and discuss existing and novel therapeutic strategies, from idea to implementation, and from bench to bedside.
induced pluripotent stem cell (iPSC) --- clustered regularly interspaced short palindromic repeats (CRISPR) --- homology-directed repair (HDR) --- Enhanced S-Cone Syndrome (ESCS) --- NR2E3 --- AAV --- retina --- gene therapy --- dual AAV --- gold nanoparticles --- DNA-wrapped gold nanoparticles --- ARPE-19 cells --- retinal pigment epithelium --- clathrin-coated vesicles --- endosomal trafficking --- retinitis pigmentosa --- autosomal dominant --- G56R --- putative dominant negative effect --- gapmer antisense oligonucleotides --- allele-specific knockdown --- Leber congenital amaurosis and allied retinal ciliopathies --- CEP290 --- Flanders founder c.4723A > --- T nonsense mutation --- Cilia elongation --- spontaneous nonsense correction --- AON-mediated exon skipping --- microRNA --- photoreceptors --- rods --- cones --- bipolar cells --- Müller glia --- retinal inherited disorders --- retinal degeneration --- antisense oligonucleotides --- Stargardt disease --- inherited retinal diseases --- splicing modulation --- RNA therapy --- ABCA4 --- iPSC-derived photoreceptor precursor cells --- cyclic GMP --- apoptosis --- necrosis --- drug delivery systems --- translational medicine --- Usher syndrome --- Leber congenital amaurosis --- RPE65 --- nonprofit --- patient registry --- translational --- protein trafficking --- protein folding --- protein degradation --- chaperones --- chaperonins --- heat shock response --- unfolded protein response --- autophagy --- therapy --- IRD --- DNA therapies --- RNA therapies --- compound therapies --- clinical trials --- Retinitis Pigmentosa GTPase Regulator --- adeno-associated viral --- Retinitis Pigmentosa (RP) --- choroideremia --- REP1 --- inherited retinal disease --- treatment --- apical polarity --- crumbs complex --- fetal retina --- PAR complex --- retinal organoids --- retinogenesis --- gene augmentation --- adeno-associated virus (AAV) --- n/a --- Müller glia
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Comprehensively covers protein subcellular localization from single-label prediction to multi-label prediction, and includes prediction strategies for virus, plant, and eukaryote species. Three machine learning tools are introduced to improve classification refinement, feature extraction, and dimensionality reduction.
Artificial intelligence. --- Probability. --- Protein transport. --- Statistics as Topic --- Computing Methodologies --- Biological Transport --- Mathematical Concepts --- Phenomena and Processes --- Health Care Evaluation Mechanisms --- Information Science --- Epidemiologic Methods --- Metabolism --- Public Health --- Investigative Techniques --- Metabolic Phenomena --- Quality of Health Care --- Health Care Quality, Access, and Evaluation --- Analytical, Diagnostic and Therapeutic Techniques and Equipment --- Environment and Public Health --- Health Care --- Probability --- Protein Transport --- Artificial Intelligence --- Human Anatomy & Physiology --- Health & Biological Sciences --- Animal Biochemistry --- Computational Intelligence --- AI (Artificial Intelligence) --- Computer Reasoning --- Computer Vision Systems --- Knowledge Acquisition (Computer) --- Knowledge Representation (Computer) --- Machine Intelligence --- Acquisition, Knowledge (Computer) --- Computer Vision System --- Intelligence, Artificial --- Intelligence, Computational --- Intelligence, Machine --- Knowledge Representations (Computer) --- Reasoning, Computer --- Representation, Knowledge (Computer) --- System, Computer Vision --- Systems, Computer Vision --- Vision System, Computer --- Vision Systems, Computer --- Heuristics --- Protein Localization Processes, Cellular --- Protein Sorting --- Protein Trafficking --- Cellular Protein Targeting --- Gated Protein Transport --- Protein Targeting, Cellular --- Protein Translocation --- Transmembrane Protein Transport --- Vesicular Protein Transport --- Protein Transport, Gated --- Protein Transport, Transmembrane --- Protein Transport, Vesicular --- Traffickings, Protein --- Protein Sorting Signals --- Vesicular Transport Proteins --- Probabilities --- Community-Based Distribution --- Contraceptive Distribution --- Delivery of Healthcare --- Dental Care Delivery --- Distribution, Non-Clinical --- Distribution, Nonclinical --- Distributional Activities --- Healthcare --- Healthcare Delivery --- Healthcare Systems --- Non-Clinical Distribution --- Nonclinical Distribution --- Delivery of Dental Care --- Health Care Delivery --- Health Care Systems --- Activities, Distributional --- Activity, Distributional --- Care, Health --- Community Based Distribution --- Community-Based Distributions --- Contraceptive Distributions --- Deliveries, Healthcare --- Delivery, Dental Care --- Delivery, Health Care --- Delivery, Healthcare --- Distribution, Community-Based --- Distribution, Contraceptive --- Distribution, Non Clinical --- Distributional Activity --- Distributions, Community-Based --- Distributions, Contraceptive --- Distributions, Non-Clinical --- Distributions, Nonclinical --- Health Care System --- Healthcare Deliveries --- Healthcare System --- Non Clinical Distribution --- Non-Clinical Distributions --- Nonclinical Distributions --- System, Health Care --- System, Healthcare --- Systems, Health Care --- Systems, Healthcare --- Healthcare Quality, Access, and Evaluation --- Pharmacy Audit --- Quality of Care --- Quality of Healthcare --- Audit, Pharmacy --- Care Quality --- Health Care Quality --- Healthcare Quality --- Pharmacy Audits --- Metabolic Phenomenon --- Metabolic Process --- Metabolism Concepts --- Metabolism Phenomena --- Process, Metabolic --- Processes, Metabolic --- Anabolism --- Catabolism --- Metabolic Concepts --- Metabolic Processes --- Concept, Metabolic --- Concept, Metabolism --- Concepts, Metabolic --- Concepts, Metabolism --- Metabolic Concept --- Metabolism Concept --- Phenomena, Metabolic --- Phenomena, Metabolism --- Phenomenon, Metabolic --- Investigative Technics --- Investigative Technic --- Investigative Technique --- Technic, Investigative --- Technics, Investigative --- Technique, Investigative --- Techniques, Investigative --- Community Health --- Environment, Preventive Medicine & Public Health --- Environment, Preventive Medicine and Public Health --- Health, Community --- Health, Public --- Preventive Medicine --- Education, Public Health Professional --- Epidemiologic Method --- Epidemiological Methods --- Methods, Epidemiologic --- Epidemiological Method --- Method, Epidemiologic --- Method, Epidemiological --- Methods, Epidemiological --- Epidemiology --- Information Sciences --- Science, Information --- Sciences, Information --- Healthcare Evaluation Mechanisms --- Evaluation Mechanism, Healthcare --- Evaluation Mechanisms, Healthcare --- Healthcare Evaluation Mechanism --- Mechanism, Healthcare Evaluation --- Mechanisms, Healthcare Evaluation --- Concept, Mathematical --- Concepts, Mathematical --- Mathematical Concept --- Biologic Transport --- Transport, Biological --- Transport, Biologic --- Transport Vesicles --- Membrane Transport Proteins --- High Performance Computing --- Methodologies, Computing --- Computing Methodology --- Computing, High Performance --- Methodology, Computing --- Performance Computing, High --- Area Analysis --- Estimation Technics --- Estimation Techniques --- Indirect Estimation Technics --- Indirect Estimation Techniques --- Multiple Classification Analysis --- Service Statistics --- Statistical Study --- Statistics, Service --- Tables and Charts as Topic --- Analyses, Area --- Analyses, Multiple Classification --- Area Analyses --- Classification Analyses, Multiple --- Classification Analysis, Multiple --- Estimation Technic, Indirect --- Estimation Technics, Indirect --- Estimation Technique --- Estimation Technique, Indirect --- Estimation Techniques, Indirect --- Indirect Estimation Technic --- Indirect Estimation Technique --- Multiple Classification Analyses --- Statistical Studies --- Studies, Statistical --- Study, Statistical --- Technic, Indirect Estimation --- Technics, Estimation --- Technics, Indirect Estimation --- Technique, Estimation --- Technique, Indirect Estimation --- Techniques, Estimation --- Techniques, Indirect Estimation --- methods --- Proteins --- Machine learning. --- Physiological transport --- Data processing. --- Learning, Machine --- Artificial intelligence --- Machine theory --- Proteids --- Biomolecules --- Polypeptides --- Proteomics --- Bioinformatics. --- Computer Science. --- Proteomics.
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