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Advanced delivery and therapeutic applications of RNAi [[electronic resource] /] / edited by Kun Cheng, Ram I. Mahato
| Advanced delivery and therapeutic applications of RNAi [[electronic resource] /] / edited by Kun Cheng, Ram I. Mahato |
| Edizione | [2nd ed.] |
| Pubbl/distr/stampa | Chichester, West Sussex, : John Wiley & Sons Inc., 2013 |
| Descrizione fisica | 1 online resource (535 p.) |
| Disciplina | 572.8/8 |
| Altri autori (Persone) |
ChengKun
MahatoRam I |
| Soggetto topico |
RNA - Therapeutic use
Small interfering RNA - Therapeutic use |
| ISBN |
1-118-61073-3
1-118-61074-1 1-299-46528-5 1-118-61075-X |
| Classificazione | SCI013040 |
| Formato | Materiale a stampa  |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto |
Advanced Delivery and Therapeutic Applications of RNAi; Contents; Preface; Contributors; About the Editors; Part 1: Introduction and Basics of RNAi; 1 Mechanisms and Barriers to RNAi Delivery; 1.1 Introduction; 1.2 Barriers to Systemic RNAi Delivery; 1.3 Rational Design to Improve RNAi Efficacy; 1.4 Chemical Modifications to Enhance siRNA Stability and Reduce Immune Response; 1.5 Cellular Uptake and Intracellular Release of siRNA; 1.6 Combinatorial Targeting for Targeted RNAi Delivery; 1.7 Cell-Specific Aptamer-Functionalized Nanocarriers for RNAi Delivery
1.8 The Clinical Development and Challenges of siRNAs Therapeutics1.9 Conclusion and Perspectives; References; 2 Analysis of siRNA Delivery Using Various Methodologies; 2.1 Introduction; 2.2 Checkpoints for Analyzing siRNA Delivery; 2.2.1 Circulation Checkpoint; 2.2.2 Organ or Tissue Checkpoint; 2.2.3 Cellular Checkpoint; 2.2.4 RISC Checkpoint; 2.2.5 Target mRNA Knockdown (Indirect Checkpoint); 2.2.6 Protein and Outcome (Indirect Checkpoint); 2.2.7 Safety (Indirect Checkpoint); 2.3 Methods for Analysis of siRNA; 2.3.1 General Considerations; 2.3.2 Hybridization-Based (Non-Imaging) Methods 2.3.3 Non-Hybridization-Based (Non-Imaging) Methods2.3.4 Imaging-Based (Non-Hybridization) Methods; 2.3.5 Imaging-Based (Hybridization) Methods; 2.4 Case Study for siRNA Delivery Analysis; References; 3 Challenges and Opportunities in Bringing RNAi Technologies from Bench to Bed; 3.1 Introduction; 3.2 RNAi Mediator (siRNA or shRNA); 3.2.1 siRNA; 3.2.2 Vector-derived shRNA; 3.2.3 miRNAs; 3.3 Safety Issues of RNAi Mediators; 3.3.1 Immune Stimulation; 3.3.2 RNAi Overexpression; 3.4 Efficacy of RNAi Mediators; 3.4.1 Therapeutic Response; 3.5 RNAi Mediators in Clinical Trials; 3.6 Conclusion References4 Nonclinical Safety Assessments and Clinical Pharmacokinetics for Oligonucleotide Therapeutics: A Regulatory Perspective; 4.1 Introduction; 4.2 Unique Properties of Oligonucleotide-based Therapeutics; 4.3 Regulation of Oligonucleotide-Based Therapeutics; 4.3.1 Submission to the FDA; 4.3.2 Review Process for Non-clinical Studies; 4.3.3 Regulatory Issues; 4.3.4 Clinical Pharmacokinetics; 4.4 Conclusion; Disclaimer; Appendix; References; 5 Role of Promoters and MicroRNA Backbone for Efficient Gene Silencing; 5.1 Introduction; 5.2 Promoters for shRNA Expression 5.2.1 Constitutive Promoters5.2.2 Inducible Promoters; 5.2.3 Site Specific Promoters; 5.3 miRNA-based shRNAs; 5.3.1 miRNA-based shRNA Enhances Gene Silencing; 5.3.2 miRNA-based shRNA Reduces Toxicities; 5.3.3 Application of miRNA-based shRNA for Combination Gene Therapy; 5.4 Concluding Remarks; References; Part 2: RNAi Delivery Strategies; 6 Bioconjugation of siRNA for Site-specific Delivery; 6.1 Introduction; 6.2 Conjugation Strategy; 6.2.1 RNA Chemical Modification; 6.2.2 Site of Conjugation; 6.2.3 Conjugation Chemistry; 6.3 Bioconjugates for Site-specific Delivery 6.3.1 Antibody-siRNA Bioconjugates |
| Record Nr. | UNINA-9910139008603321 |
| Chichester, West Sussex, : John Wiley & Sons Inc., 2013 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
Advanced delivery and therapeutic applications of RNAi / / edited by Kun Cheng, Ram I. Mahato
| Advanced delivery and therapeutic applications of RNAi / / edited by Kun Cheng, Ram I. Mahato |
| Edizione | [2nd ed.] |
| Pubbl/distr/stampa | Chichester, West Sussex, : John Wiley & Sons Inc., 2013 |
| Descrizione fisica | 1 online resource (535 p.) |
| Disciplina | 572.8/8 |
| Altri autori (Persone) |
ChengKun
MahatoRam I |
| Soggetto topico |
RNA - Therapeutic use
Small interfering RNA - Therapeutic use |
| ISBN |
1-118-61073-3
1-118-61074-1 1-299-46528-5 1-118-61075-X |
| Classificazione | SCI013040 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto |
Advanced Delivery and Therapeutic Applications of RNAi; Contents; Preface; Contributors; About the Editors; Part 1: Introduction and Basics of RNAi; 1 Mechanisms and Barriers to RNAi Delivery; 1.1 Introduction; 1.2 Barriers to Systemic RNAi Delivery; 1.3 Rational Design to Improve RNAi Efficacy; 1.4 Chemical Modifications to Enhance siRNA Stability and Reduce Immune Response; 1.5 Cellular Uptake and Intracellular Release of siRNA; 1.6 Combinatorial Targeting for Targeted RNAi Delivery; 1.7 Cell-Specific Aptamer-Functionalized Nanocarriers for RNAi Delivery
1.8 The Clinical Development and Challenges of siRNAs Therapeutics1.9 Conclusion and Perspectives; References; 2 Analysis of siRNA Delivery Using Various Methodologies; 2.1 Introduction; 2.2 Checkpoints for Analyzing siRNA Delivery; 2.2.1 Circulation Checkpoint; 2.2.2 Organ or Tissue Checkpoint; 2.2.3 Cellular Checkpoint; 2.2.4 RISC Checkpoint; 2.2.5 Target mRNA Knockdown (Indirect Checkpoint); 2.2.6 Protein and Outcome (Indirect Checkpoint); 2.2.7 Safety (Indirect Checkpoint); 2.3 Methods for Analysis of siRNA; 2.3.1 General Considerations; 2.3.2 Hybridization-Based (Non-Imaging) Methods 2.3.3 Non-Hybridization-Based (Non-Imaging) Methods2.3.4 Imaging-Based (Non-Hybridization) Methods; 2.3.5 Imaging-Based (Hybridization) Methods; 2.4 Case Study for siRNA Delivery Analysis; References; 3 Challenges and Opportunities in Bringing RNAi Technologies from Bench to Bed; 3.1 Introduction; 3.2 RNAi Mediator (siRNA or shRNA); 3.2.1 siRNA; 3.2.2 Vector-derived shRNA; 3.2.3 miRNAs; 3.3 Safety Issues of RNAi Mediators; 3.3.1 Immune Stimulation; 3.3.2 RNAi Overexpression; 3.4 Efficacy of RNAi Mediators; 3.4.1 Therapeutic Response; 3.5 RNAi Mediators in Clinical Trials; 3.6 Conclusion References4 Nonclinical Safety Assessments and Clinical Pharmacokinetics for Oligonucleotide Therapeutics: A Regulatory Perspective; 4.1 Introduction; 4.2 Unique Properties of Oligonucleotide-based Therapeutics; 4.3 Regulation of Oligonucleotide-Based Therapeutics; 4.3.1 Submission to the FDA; 4.3.2 Review Process for Non-clinical Studies; 4.3.3 Regulatory Issues; 4.3.4 Clinical Pharmacokinetics; 4.4 Conclusion; Disclaimer; Appendix; References; 5 Role of Promoters and MicroRNA Backbone for Efficient Gene Silencing; 5.1 Introduction; 5.2 Promoters for shRNA Expression 5.2.1 Constitutive Promoters5.2.2 Inducible Promoters; 5.2.3 Site Specific Promoters; 5.3 miRNA-based shRNAs; 5.3.1 miRNA-based shRNA Enhances Gene Silencing; 5.3.2 miRNA-based shRNA Reduces Toxicities; 5.3.3 Application of miRNA-based shRNA for Combination Gene Therapy; 5.4 Concluding Remarks; References; Part 2: RNAi Delivery Strategies; 6 Bioconjugation of siRNA for Site-specific Delivery; 6.1 Introduction; 6.2 Conjugation Strategy; 6.2.1 RNA Chemical Modification; 6.2.2 Site of Conjugation; 6.2.3 Conjugation Chemistry; 6.3 Bioconjugates for Site-specific Delivery 6.3.1 Antibody-siRNA Bioconjugates |
| Record Nr. | UNINA-9910815366903321 |
| Chichester, West Sussex, : John Wiley & Sons Inc., 2013 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||