Recent Advances in Molecular and Translational Medicine.

Format:

Book

Author/Creator:

Chutipongtanate, Somchai.

Contributor:

Hongeng, Suradej.

Language:

English

Subjects (All):

Molecular biology--Periodicals.

Molecular biology.

Physical Description:

1 online resource (253 pages)

Edition:

1st ed.

Place of Publication:

Singapore : Bentham Science Publishers, 2021.

Summary:

Precision medicine is a disruptive innovation with a fast-evolving pace in the healthcare ecosystem. Precision medicine enables precise diagnosis and targeted treatment by considering individual variability in the abnormalities of causative genes and molecular drivers behind biochemical mechanisms. A vast amount of data created by advanced omics technologies is a foundation of precision medicine's success, and the implications of the findings from these technologies can potentially improve clinical outcomes. Recent Advances in Molecular and Translational Medicine: Updates in Precision Medicine presents essential information of molecular and translational research in precision medicine, with a specific focus on pediatrics. This book provides an accessible introduction to omics technologies, gives a detailed explanation of bioinformatics workflows to interpret high-throughput omics profiles for molecular diagnosis, and collects some of the cutting-edge research for precise therapeutics. Contributions to the book have been provided by experts in biomedical engineering and clinical practice, thus, bringing an informed perspective to the reader on each topic. The book is a valuable resource for postgraduate students, researchers, data scientists and clinicians interested in precision medicine, as well as researchers in the field of genetics and pediatrics who are interested in understanding the role of precision medicine in clinical practice.

Contents:

Intro

Table of Content

Title

BENTHAM SCIENCE PUBLISHERS LTD.

End User License Agreement (for non-institutional, personal use)

Usage Rules:

Disclaimer:

Limitation of Liability:

General:

FOREWORD

PREFACE

List of Contributors

Translational Research in Pediatric Precision Medicine

Abstract

INTRODUCTION

THE CONCEPTS OF TRANSLATIONAL RESEARCH AND PRECISION MEDICINE

Translational Research

Precision Medicine

DEVELOPING CAPACITY OF TRANSLATIONAL RESEARCH IN PEDIATRIC PRECISION MEDICINE

Biobanking

"Omics" Technologies

Data Management and Resources

FUTURE PROSPECTS AND CONCLUDING REMARKS

CONSENT FOR PUBLICATION

CONFLICT OF INTEREST

ACKNOWLEDGMENTS

REFERENCES

Bioinformatics in Precision Medicine

GENERAL CONCEPTS

Genomics

Transcriptomics

Proteomics

Post-Translational Modifications (PTM)

Metabolomics

Metagenomics

BIOINFORMATICS

The Concept of Molecular Signature

Genomic Workflow

Transcriptomic Workflow

Quality Control (QC)

Read Mapping (or Alignment)

Read Count Normalization

Differential Expression and Functional Interpretation

Unsupervised Clustering

Volcano Plot

Enrichment Analysis

Proteomic Workflow

Metabolomic Workflow

Targeted

Untargeted

Data Analysis

MetaboAnalyst

Metagenomic Workflow

MULTI-OMICS &amp

CONCLUDING REMARKS

Artificial Intelligence in Medicine

DEFINITION OF MACHINE LEARNING

Supervised Learning Algorithms

Unsupervised Learning Algorithms

Deep learning

ARTIFICIAL INTELLIGENCE FOR CLINICIANS

Computer Vision

Dermatology

Radiology

Ophthalmology

Pathology

Pulmonary

Natural Language Processing.

LIMITATIONS AND CHALLENGES

CONCLUSION

Using Next Generation Sequencing for Diagnosis of Suspected Genetic Disorders

VARIANT, VARIANT CLASSIFICATION AND INTERPRETATION

APPROACHES OF CLINICAL NGS

SECONDARY FINDINGS

DIAGNOSTIC AND CLINICAL UTILITY OF CLINICAL NGS

ETHICAL ISSUES AND CONDLUDING REMARKS

ACKNOWLEDGEMENTS

Pharmacogenomics and Perioperative Translational Medicine

Developmental Pharmacogenomics Concept

What is Perioperative Medicine?

RECENT TRENDS IN PERIOPERATIVE TRANSLATION MEDICINE

Opioids

Fentanyl

Codeine

Morphine

Oxycodone

Tramadol

Methadone

Hydrocodone and Hydromorphone

Inhalation Agents

Intravenous Induction Agents

Propofol

Ketamine

Neuromuscular Blocking Agents

Succinylcholine

Rocuronium

Sedative Drugs

Diazepam

Midazolam

Proteomics: Moving Toward Precision Medicine

PROTEOMIC APPROACHES

Bottom-Up Proteomics vs. Top-Down Proteomics

Discovery Proteomics vs. Targeted Proteomics

Quantitative Proteomics

DOWNSTREAM DATA ANALYSIS

Protein and Peptide Identification

Preprocessing

Normalization

Missing Value Imputation

Data Analysis and Visualization

Differential Expression Analysis

Heatmap

Machine Learning (ML)

Surrogate Variable Analysis (SVA) &amp

Co-variates Calculation

Overrepresentation Enrichment Analysis

Protein Network Analysis

PROTEOMIC APPLICATIONS IN PRECISION MEDICINE

Biomarkers for Precise Diagnosis.

Mechanistic Insights for Precision Treatment with Focus on Post-Translationally Modified Proteins

CONCLUDING REMARKS AND FUTURE PROSPECTS

Metabolomics and Precision Medicine

THE CONCEPT OF METABOLOMIC SIGNATURE

METABOLOMIC APPROACHES

Targeted vs. Untargeted Analysis

Data Acquisition by NMR, GC-MS, or LC-MS Platforms

DATA PREPROCESSING AND ANALYSIS

Data Preprocessing

Data Filtering

Transformation

Visualizing Data Distribution

Fold Change Analysis

Significance Test (Parametric - T-Test

Non-Parametric - Wilcoxon)

Principal Component Analysis (PCA)

Partial Least Squares - Discriminant Analysis (PLS-DA)

Cluster Analysis

Hierarchical Clustering

Partitional Clustering

Machine Learning Classification

Pathway Enrichment Analysis

POTENTIAL APPLICATIONS OF METABOLOMICS IN PRECISION MEDICINE

Genetically Modified Hematopoietic Stem Cells for Gene Therapy in Beta-Thalassemia: Power of Viral Vector Towards Clinical Application

CELL AND GENE THERAPY FOR THE TREATMENT OF β-THALASSEMIA

β-Globin Vector Development and Preclinical Evaluation

Clinical Trial in β-Thalassemia Patients

Molecular Therapeutic Approach Focusing on Kinases

BIOLOGY OF KINASE WORLD ("KINOME")

DISEASE BIOLOGY AND DYSFUNCTIONAL KINOME ("YIN AND YANG PHENOMENON")

KINOME THERAPEUTICS ("THERAPEUTIC'S HOLY GRAIL").

KINOME THERAPEUTICS IN PEDIATRIC DISEASES

Membrane Attacking Peptides: A New Involving Cancer Therapy

NORMAL CELLS VS CANCER CELLS

Membrane Components

Stability and Molecular Transports

Membrane and Peptide Interaction

CLINICAL TRIALS IN CANCER THERAPEUTIC PEPTIDES

CONCLUDING REMARKS AND FUTURE PERSPECTIVES

Chimeric Antigen Receptor (CAR) T Cell for Pediatric Solid Tumors: The Next Frontier in Cancer Treatment

THE STATE OF IMMUNOTHERAPY FOR PEDIATRIC SOLID TUMORS

Concept and Evolution of CARs

General Principle of CAR Design

The Single-chain Variable Fragment

Hinge/Spacer Domains

Transmembrane Domain

Intracellular Domain

RECENT CLINICAL ADVANCES IN CAR T CELL THERAPY FOR PEDIATRIC SOLID TUMORS

Sarcoma

Brain Tumor

Neuroblastoma

ROADBLOCKS FOR SUCCESSFUL CAR T CELL THERAPIES

Safety Concerns: The Side Effect Profile and Toxicity

T Cell Trafficking

Tumor Microenvironment

Immune Suppressive Cells

Inhibitory Signals in the TME

Metabolic Challenges and T Cell Dysfunction

MOVING FORWARD CAR T CELLS FROM THE BENCH TO THE BEDSIDE: COMMERCIALIZATION OF CAR T THERAPY

FUTURE PROSPECTS: THE NEXT GENERATION OF CARS

Combination Strategies

Empowering CAR to withstand the TME

Moving Toward the Universal CARs: Genome Editing and SUPRA CAR

Hematopoietic Stem Cell Transplant in Inherited Genetic Diseases

HSCT PROCEDURE AS PRECISE TREATMENT

CLINICAL EXPERIENCES

Thalassemia.

Inherited Bone Marrow Failure Syndromes

Primary Immunodeficiency Diseases

REFERENCES.

Notes:

Includes bibliographical references.

Description based on publisher supplied metadata and other sources.

ISBN:

9789815036756

9815036750

OCLC:

1293254228