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Book
Oligonucleotide, Therapy, and Applications
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Year: 2022 Publisher: Basel MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

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.


Book
Oligonucleotide, Therapy, and Applications
Authors: ---
Year: 2022 Publisher: Basel MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

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.


Book
Oligonucleotide, Therapy, and Applications
Authors: ---
Year: 2022 Publisher: Basel MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

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.


Book
Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function
Authors: ---
Year: 2021 Publisher: Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

This compilation focuses on recent advances in the molecular and cellular understandingof neuromuscular biology, and the treatment of neuromuscular disease.These advances are at the forefront of modern molecular methodologies, oftenintegrating across wet-lab cell and tissue models, dry-lab computational approaches,and clinical studies. The continuing development and application ofmultiomics methods offer particular challenges and opportunities in the field,not least in the potential for personalized medicine.

Keywords

Research & information: general --- LMNA --- Emery–Dreifuss muscular dystrophy --- Omics --- ALS --- MND --- ALS variants --- genotype–phenotype --- ALS genes --- FSHD --- DUX4 --- transcription --- muscle --- regulation --- spinal muscular atrophy --- adult patients --- disease heterogeneity --- Nusinersen --- disease modifiers --- functional outcomes --- biomarkers --- epigenetic changes --- -omics approaches --- oxidative stress --- mitochondria dysfunction --- axonal transport --- autophagy --- endocytosis --- secretion --- excitotoxicity --- RNA metabolism --- Duchenne muscular dystrophy (DMD) --- exon-skipping therapies --- next-generation sequencing (NGS) --- Sanger sequencing --- multiplex ligation probe amplification (MLPA) --- multiplex polymerase chain reaction (PCR) --- comparative genomic hybridization array (CGH) --- viltolarsen --- eteplirsen --- golodirsen --- rheumatoid arthritis --- SNP --- DMARD --- methotrexate --- pharmacogenomics --- Duchenne muscular dystrophy --- pharmacodynamic biomarkers --- prednisone --- deflazacort --- glucocorticoids --- corticosteroids --- safety --- neuromuscular diseases --- translational research --- disease models --- precision medicine --- miRNA --- proteomics --- calprotectin --- dystrophy --- Becker muscular dystrophy --- dystrophinopathy --- genotype-phenotype correlations --- Canadian Neuromuscular Disease Registry --- reading frame rule --- dystrophin --- multiple logistic regression analysis --- exon skipping therapy --- Amyotrophic Lateral Sclerosis --- machine learning --- genome-wide association studies --- GWAS --- genomics --- ALS pathology --- gene prioritization --- AAV --- genetic neuromuscular disorders --- gene therapy --- clinical trials --- toxicity --- SMA --- DMD --- XLMTM --- facioscapulohumeral dystrophy --- TALEN --- CRISPR-Cas9 --- gene editing --- polyadenylation --- D4Z4 --- duchenne muscular dystrophy (DMD) --- becker muscular dystrophy (BMD) --- exon skipping --- skip-equivalent deletions --- n/a --- Emery-Dreifuss muscular dystrophy --- genotype-phenotype


Book
Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function
Authors: ---
Year: 2021 Publisher: Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

This compilation focuses on recent advances in the molecular and cellular understandingof neuromuscular biology, and the treatment of neuromuscular disease.These advances are at the forefront of modern molecular methodologies, oftenintegrating across wet-lab cell and tissue models, dry-lab computational approaches,and clinical studies. The continuing development and application ofmultiomics methods offer particular challenges and opportunities in the field,not least in the potential for personalized medicine.

Keywords

LMNA --- Emery–Dreifuss muscular dystrophy --- Omics --- ALS --- MND --- ALS variants --- genotype–phenotype --- ALS genes --- FSHD --- DUX4 --- transcription --- muscle --- regulation --- spinal muscular atrophy --- adult patients --- disease heterogeneity --- Nusinersen --- disease modifiers --- functional outcomes --- biomarkers --- epigenetic changes --- -omics approaches --- oxidative stress --- mitochondria dysfunction --- axonal transport --- autophagy --- endocytosis --- secretion --- excitotoxicity --- RNA metabolism --- Duchenne muscular dystrophy (DMD) --- exon-skipping therapies --- next-generation sequencing (NGS) --- Sanger sequencing --- multiplex ligation probe amplification (MLPA) --- multiplex polymerase chain reaction (PCR) --- comparative genomic hybridization array (CGH) --- viltolarsen --- eteplirsen --- golodirsen --- rheumatoid arthritis --- SNP --- DMARD --- methotrexate --- pharmacogenomics --- Duchenne muscular dystrophy --- pharmacodynamic biomarkers --- prednisone --- deflazacort --- glucocorticoids --- corticosteroids --- safety --- neuromuscular diseases --- translational research --- disease models --- precision medicine --- miRNA --- proteomics --- calprotectin --- dystrophy --- Becker muscular dystrophy --- dystrophinopathy --- genotype-phenotype correlations --- Canadian Neuromuscular Disease Registry --- reading frame rule --- dystrophin --- multiple logistic regression analysis --- exon skipping therapy --- Amyotrophic Lateral Sclerosis --- machine learning --- genome-wide association studies --- GWAS --- genomics --- ALS pathology --- gene prioritization --- AAV --- genetic neuromuscular disorders --- gene therapy --- clinical trials --- toxicity --- SMA --- DMD --- XLMTM --- facioscapulohumeral dystrophy --- TALEN --- CRISPR-Cas9 --- gene editing --- polyadenylation --- D4Z4 --- duchenne muscular dystrophy (DMD) --- becker muscular dystrophy (BMD) --- exon skipping --- skip-equivalent deletions --- n/a --- Emery-Dreifuss muscular dystrophy --- genotype-phenotype


Book
Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function
Authors: ---
Year: 2021 Publisher: Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute

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Bookmark

Abstract

This compilation focuses on recent advances in the molecular and cellular understandingof neuromuscular biology, and the treatment of neuromuscular disease.These advances are at the forefront of modern molecular methodologies, oftenintegrating across wet-lab cell and tissue models, dry-lab computational approaches,and clinical studies. The continuing development and application ofmultiomics methods offer particular challenges and opportunities in the field,not least in the potential for personalized medicine.

Keywords

Research & information: general --- LMNA --- Emery-Dreifuss muscular dystrophy --- Omics --- ALS --- MND --- ALS variants --- genotype-phenotype --- ALS genes --- FSHD --- DUX4 --- transcription --- muscle --- regulation --- spinal muscular atrophy --- adult patients --- disease heterogeneity --- Nusinersen --- disease modifiers --- functional outcomes --- biomarkers --- epigenetic changes --- -omics approaches --- oxidative stress --- mitochondria dysfunction --- axonal transport --- autophagy --- endocytosis --- secretion --- excitotoxicity --- RNA metabolism --- Duchenne muscular dystrophy (DMD) --- exon-skipping therapies --- next-generation sequencing (NGS) --- Sanger sequencing --- multiplex ligation probe amplification (MLPA) --- multiplex polymerase chain reaction (PCR) --- comparative genomic hybridization array (CGH) --- viltolarsen --- eteplirsen --- golodirsen --- rheumatoid arthritis --- SNP --- DMARD --- methotrexate --- pharmacogenomics --- Duchenne muscular dystrophy --- pharmacodynamic biomarkers --- prednisone --- deflazacort --- glucocorticoids --- corticosteroids --- safety --- neuromuscular diseases --- translational research --- disease models --- precision medicine --- miRNA --- proteomics --- calprotectin --- dystrophy --- Becker muscular dystrophy --- dystrophinopathy --- genotype-phenotype correlations --- Canadian Neuromuscular Disease Registry --- reading frame rule --- dystrophin --- multiple logistic regression analysis --- exon skipping therapy --- Amyotrophic Lateral Sclerosis --- machine learning --- genome-wide association studies --- GWAS --- genomics --- ALS pathology --- gene prioritization --- AAV --- genetic neuromuscular disorders --- gene therapy --- clinical trials --- toxicity --- SMA --- DMD --- XLMTM --- facioscapulohumeral dystrophy --- TALEN --- CRISPR-Cas9 --- gene editing --- polyadenylation --- D4Z4 --- duchenne muscular dystrophy (DMD) --- becker muscular dystrophy (BMD) --- exon skipping --- skip-equivalent deletions


Book
Peptide Nucleic Acids : Applications in Biomedical Sciences
Author:
Year: 2020 Publisher: Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

This book compiles four review articles and four research papers that highlight recent developments in the field of peptide nucleic acid (PNA) chemistry and biomedicine. The review articles encompass a variety of fields related to PNA, emphasizing the versatility of this DNA mimic. Two excellent reviews detail the use of PNA for molecular diagnostics of miRNAs and genetic point mutations (SNPs). Another review provides a comprehensive analysis of the various approaches for gene editing using chemically modified PNA molecules. Lastly, PNA molecules are elegantly described as effective (antisense) antimicrobial agents in the final review. The high binding affinity of PNA to complementary DNA and RNA is highlighted in three research articles. Two articles show how PNA molecules act as splice modulating and RNA masking molecules, separately. In another contribution, the high affinity and achiral characteristics of PNAs are used to developed a stable L-DNA-based catalytic hairpin assembly. Lastly, chemically-modified PNA molecules are shown to be superior probes for SNP detection. Altogether, these studies illustrate how PNA molecules may be useful for a variety of biomedical applications as either therapeutic or diagnostic agents.


Book
Peptide Nucleic Acids : Applications in Biomedical Sciences
Author:
Year: 2020 Publisher: Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

This book compiles four review articles and four research papers that highlight recent developments in the field of peptide nucleic acid (PNA) chemistry and biomedicine. The review articles encompass a variety of fields related to PNA, emphasizing the versatility of this DNA mimic. Two excellent reviews detail the use of PNA for molecular diagnostics of miRNAs and genetic point mutations (SNPs). Another review provides a comprehensive analysis of the various approaches for gene editing using chemically modified PNA molecules. Lastly, PNA molecules are elegantly described as effective (antisense) antimicrobial agents in the final review. The high binding affinity of PNA to complementary DNA and RNA is highlighted in three research articles. Two articles show how PNA molecules act as splice modulating and RNA masking molecules, separately. In another contribution, the high affinity and achiral characteristics of PNAs are used to developed a stable L-DNA-based catalytic hairpin assembly. Lastly, chemically-modified PNA molecules are shown to be superior probes for SNP detection. Altogether, these studies illustrate how PNA molecules may be useful for a variety of biomedical applications as either therapeutic or diagnostic agents.


Book
Peptide Nucleic Acids : Applications in Biomedical Sciences
Author:
Year: 2020 Publisher: Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

This book compiles four review articles and four research papers that highlight recent developments in the field of peptide nucleic acid (PNA) chemistry and biomedicine. The review articles encompass a variety of fields related to PNA, emphasizing the versatility of this DNA mimic. Two excellent reviews detail the use of PNA for molecular diagnostics of miRNAs and genetic point mutations (SNPs). Another review provides a comprehensive analysis of the various approaches for gene editing using chemically modified PNA molecules. Lastly, PNA molecules are elegantly described as effective (antisense) antimicrobial agents in the final review. The high binding affinity of PNA to complementary DNA and RNA is highlighted in three research articles. Two articles show how PNA molecules act as splice modulating and RNA masking molecules, separately. In another contribution, the high affinity and achiral characteristics of PNAs are used to developed a stable L-DNA-based catalytic hairpin assembly. Lastly, chemically-modified PNA molecules are shown to be superior probes for SNP detection. Altogether, these studies illustrate how PNA molecules may be useful for a variety of biomedical applications as either therapeutic or diagnostic agents.


Book
Carotenoids
Author:
ISBN: 3039218654 3039218646 Year: 2019 Publisher: MDPI - Multidisciplinary Digital Publishing Institute

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Abstract

Carotenoids are a group of natural pigments, consisting of more than 750 compounds. They are mostly yellow, orange, or red in color, due to the system of conjugated double bonds. This structural element is also responsible for the good antioxidant properties of many carotenoids. Carotenoids have shown numerous biological activities (not only as provitamin A), e.g., preventive properties of fruits and vegetables. As lipophilic compounds, their uptake and storage in the body are dependent on various conditions. In vitro and in vivo data showed stimulating and inhibitory effects of matrix compounds on bioaccessibility and bioavailability of carotenoids.

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