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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.
Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells
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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.
Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells
Choose an application
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.
Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells
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Oligonucleotides --- Oligonucleotides. --- Oligonucleotides, Antisense. --- Anti-Sense Oligonucleotides --- Antisense Oligonucleotides --- Anti Sense Oligonucleotides --- Oligonucleotides, Anti-Sense --- Antisense Elements (Genetics). --- Nucleic Acids. --- Nucleotides --- Morpholinos --- Anti-Sense Oligonucleotide --- Antisense Oligonucleotide --- Anti Sense Oligonucleotide --- Oligonucleotide, Anti-Sense --- Oligonucleotide, Antisense --- Oligonucleotide
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Oligonucleotides (ON) constitute a new group of molecular agents, the object of significant interest due to their potential value as drugs for diagnostic and therapeutic applications. Their special interest derives from the intrinsic characteristics of ONs: a) ONs are informative agents, a property that derives from the order in which the nucleotides of each particular ON are arranged; b) ONs can act as ligands (ASO, TFO, aptamers, G-quadruplex, etc.) of complementary nucleic acid sequences (DNA or RNA) due to their high capacity to hybridize (by means of Watson and Crick or Hoogsteen links) with other nucleotide sequences, resulting in specific gene modulatory effects. However, nonspecific sequences may also be of interest, as is the case with repetitive nucleotide sequences (CpG) with adjuvant effects of vaccines; c) ONs can also rapidly evolve to achieve specific advantages of utility (targeting, stability, efficacy, toxicity, etc.) or high-sensitivity diagnostic technology (markers, analyzes, biosensors, FISH, microarrays, etc.), by chemical modification of nucleotides in any of their atoms. These properties show that ONs are first-order molecules due to their potential usefulness in practice.In this collection of research articles and review papers, we aim to highlight their therapeutic, but also diagnostic and technological utility as drugs.
Medicine --- quantum dots (QDs) --- DNAzyme --- ROS --- Amplex Red --- light-induced activity --- DNA methylation --- histone code --- microRNA --- nanoparticles --- noncoding RNA --- pulmonary arterial hypertension --- aptamer --- aptasensor --- influenza --- SERS --- virus detection --- α-synuclein --- antisense oligonucleotide --- dopamine neurotransmission --- double mutant A30P*A53T* --- motor deficits --- Parkinson’s disease --- transgenic mouse model --- G-quadruplexes --- covalent dimer construct --- anti-proliferative activity --- primary cell culture of human glioma --- antisensense oligonucleotide --- Foxp3 --- regulatory T cells --- vaccine immunogenicity --- Sporothrix schenckii --- Marfan syndrome --- fibrillin-1 --- antisense oligonucleotides --- exon skipping --- splice-switching
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Oligonucleotides (ON) constitute a new group of molecular agents, the object of significant interest due to their potential value as drugs for diagnostic and therapeutic applications. Their special interest derives from the intrinsic characteristics of ONs: a) ONs are informative agents, a property that derives from the order in which the nucleotides of each particular ON are arranged; b) ONs can act as ligands (ASO, TFO, aptamers, G-quadruplex, etc.) of complementary nucleic acid sequences (DNA or RNA) due to their high capacity to hybridize (by means of Watson and Crick or Hoogsteen links) with other nucleotide sequences, resulting in specific gene modulatory effects. However, nonspecific sequences may also be of interest, as is the case with repetitive nucleotide sequences (CpG) with adjuvant effects of vaccines; c) ONs can also rapidly evolve to achieve specific advantages of utility (targeting, stability, efficacy, toxicity, etc.) or high-sensitivity diagnostic technology (markers, analyzes, biosensors, FISH, microarrays, etc.), by chemical modification of nucleotides in any of their atoms. These properties show that ONs are first-order molecules due to their potential usefulness in practice.In this collection of research articles and review papers, we aim to highlight their therapeutic, but also diagnostic and technological utility as drugs.
Medicine --- quantum dots (QDs) --- DNAzyme --- ROS --- Amplex Red --- light-induced activity --- DNA methylation --- histone code --- microRNA --- nanoparticles --- noncoding RNA --- pulmonary arterial hypertension --- aptamer --- aptasensor --- influenza --- SERS --- virus detection --- α-synuclein --- antisense oligonucleotide --- dopamine neurotransmission --- double mutant A30P*A53T* --- motor deficits --- Parkinson’s disease --- transgenic mouse model --- G-quadruplexes --- covalent dimer construct --- anti-proliferative activity --- primary cell culture of human glioma --- antisensense oligonucleotide --- Foxp3 --- regulatory T cells --- vaccine immunogenicity --- Sporothrix schenckii --- Marfan syndrome --- fibrillin-1 --- antisense oligonucleotides --- exon skipping --- splice-switching
Choose an application
Oligonucleotides (ON) constitute a new group of molecular agents, the object of significant interest due to their potential value as drugs for diagnostic and therapeutic applications. Their special interest derives from the intrinsic characteristics of ONs: a) ONs are informative agents, a property that derives from the order in which the nucleotides of each particular ON are arranged; b) ONs can act as ligands (ASO, TFO, aptamers, G-quadruplex, etc.) of complementary nucleic acid sequences (DNA or RNA) due to their high capacity to hybridize (by means of Watson and Crick or Hoogsteen links) with other nucleotide sequences, resulting in specific gene modulatory effects. However, nonspecific sequences may also be of interest, as is the case with repetitive nucleotide sequences (CpG) with adjuvant effects of vaccines; c) ONs can also rapidly evolve to achieve specific advantages of utility (targeting, stability, efficacy, toxicity, etc.) or high-sensitivity diagnostic technology (markers, analyzes, biosensors, FISH, microarrays, etc.), by chemical modification of nucleotides in any of their atoms. These properties show that ONs are first-order molecules due to their potential usefulness in practice.In this collection of research articles and review papers, we aim to highlight their therapeutic, but also diagnostic and technological utility as drugs.
quantum dots (QDs) --- DNAzyme --- ROS --- Amplex Red --- light-induced activity --- DNA methylation --- histone code --- microRNA --- nanoparticles --- noncoding RNA --- pulmonary arterial hypertension --- aptamer --- aptasensor --- influenza --- SERS --- virus detection --- α-synuclein --- antisense oligonucleotide --- dopamine neurotransmission --- double mutant A30P*A53T* --- motor deficits --- Parkinson’s disease --- transgenic mouse model --- G-quadruplexes --- covalent dimer construct --- anti-proliferative activity --- primary cell culture of human glioma --- antisensense oligonucleotide --- Foxp3 --- regulatory T cells --- vaccine immunogenicity --- Sporothrix schenckii --- Marfan syndrome --- fibrillin-1 --- antisense oligonucleotides --- exon skipping --- splice-switching
Choose an application
In biofilms, microorganisms are able to communicate together and assemble by themselves, creating a consortium with different properties from the original free-floating microorganisms. In fact, biofilm cells bind strongly to a living or non-living surface, enclosed in a self-produced extracellular matrix that is composed of extracellular polymeric substances. One benefit of this lifestyle is the increased resistance or tolerance to antimicrobial agents (e.g., antibiotics). Hence, research on the development of alternative strategies to prevent and control biofilms is highly relevant for society in terms of human health, industry and the environment. Different approaches to prevent or control biofilms using antibiotic alternative strategies were submitted to this Special Issue.
Research & information: general --- Biology, life sciences --- Microbiology (non-medical) --- antibacterial drug --- vitamin B12 --- antisense oligonucleotides --- nucleic acid mimics --- LNA --- 2′OMe --- biocidal particles --- functionalization --- benzalkonium chloride --- Escherichia coli --- antimicrobial activity --- antimicrobial resistance --- natural alkylglycerols --- ether lipids --- 1-O-alkyl-sn-glycerols --- antibiofilm activity --- quorum sensing inhibition --- stainless steel --- polypropylene --- organic matter --- microbial resistance --- peracetic acid --- biofilms --- prosthesis-related infections --- heat shock --- ciprofloxacin --- antibacterial agents --- E. coli biofilms --- food contact surfaces --- biocontrol --- bacteriophages --- aluminum nitride --- composite --- antibacterial --- mechanical --- thermal --- cellulose acetate --- CAPE --- Candida --- antifungal --- biofilm --- apoptosis --- urinary tract devices --- probiotics --- Lactobacillus plantarum --- Lactobacillus rhamnosus --- displacement --- oral biofilm --- dental implants --- titanium implants --- antimicrobial --- surface coating --- anti-fouling --- n/a --- 2'OMe
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In biofilms, microorganisms are able to communicate together and assemble by themselves, creating a consortium with different properties from the original free-floating microorganisms. In fact, biofilm cells bind strongly to a living or non-living surface, enclosed in a self-produced extracellular matrix that is composed of extracellular polymeric substances. One benefit of this lifestyle is the increased resistance or tolerance to antimicrobial agents (e.g., antibiotics). Hence, research on the development of alternative strategies to prevent and control biofilms is highly relevant for society in terms of human health, industry and the environment. Different approaches to prevent or control biofilms using antibiotic alternative strategies were submitted to this Special Issue.
Research & information: general --- Biology, life sciences --- Microbiology (non-medical) --- antibacterial drug --- vitamin B12 --- antisense oligonucleotides --- nucleic acid mimics --- LNA --- 2′OMe --- biocidal particles --- functionalization --- benzalkonium chloride --- Escherichia coli --- antimicrobial activity --- antimicrobial resistance --- natural alkylglycerols --- ether lipids --- 1-O-alkyl-sn-glycerols --- antibiofilm activity --- quorum sensing inhibition --- stainless steel --- polypropylene --- organic matter --- microbial resistance --- peracetic acid --- biofilms --- prosthesis-related infections --- heat shock --- ciprofloxacin --- antibacterial agents --- E. coli biofilms --- food contact surfaces --- biocontrol --- bacteriophages --- aluminum nitride --- composite --- antibacterial --- mechanical --- thermal --- cellulose acetate --- CAPE --- Candida --- antifungal --- biofilm --- apoptosis --- urinary tract devices --- probiotics --- Lactobacillus plantarum --- Lactobacillus rhamnosus --- displacement --- oral biofilm --- dental implants --- titanium implants --- antimicrobial --- surface coating --- anti-fouling --- n/a --- 2'OMe
Choose an application
In biofilms, microorganisms are able to communicate together and assemble by themselves, creating a consortium with different properties from the original free-floating microorganisms. In fact, biofilm cells bind strongly to a living or non-living surface, enclosed in a self-produced extracellular matrix that is composed of extracellular polymeric substances. One benefit of this lifestyle is the increased resistance or tolerance to antimicrobial agents (e.g., antibiotics). Hence, research on the development of alternative strategies to prevent and control biofilms is highly relevant for society in terms of human health, industry and the environment. Different approaches to prevent or control biofilms using antibiotic alternative strategies were submitted to this Special Issue.
antibacterial drug --- vitamin B12 --- antisense oligonucleotides --- nucleic acid mimics --- LNA --- 2′OMe --- biocidal particles --- functionalization --- benzalkonium chloride --- Escherichia coli --- antimicrobial activity --- antimicrobial resistance --- natural alkylglycerols --- ether lipids --- 1-O-alkyl-sn-glycerols --- antibiofilm activity --- quorum sensing inhibition --- stainless steel --- polypropylene --- organic matter --- microbial resistance --- peracetic acid --- biofilms --- prosthesis-related infections --- heat shock --- ciprofloxacin --- antibacterial agents --- E. coli biofilms --- food contact surfaces --- biocontrol --- bacteriophages --- aluminum nitride --- composite --- antibacterial --- mechanical --- thermal --- cellulose acetate --- CAPE --- Candida --- antifungal --- biofilm --- apoptosis --- urinary tract devices --- probiotics --- Lactobacillus plantarum --- Lactobacillus rhamnosus --- displacement --- oral biofilm --- dental implants --- titanium implants --- antimicrobial --- surface coating --- anti-fouling --- n/a --- 2'OMe
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