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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
Opac: Controlla la disponibilità qui
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-9910815366903321
Chichester, West Sussex, : John Wiley & Sons Inc., 2013
Materiale a stampa
Lo trovi qui: Univ. Federico II
Opac: Controlla la disponibilità qui
MicroRNAs in toxicology and medicine [[electronic resource] /] / editor, Saura C. Sahu
MicroRNAs in toxicology and medicine [[electronic resource] /] / editor, Saura C. Sahu
Pubbl/distr/stampa Chichester, West Sussex, U.K., : John Wiley & Sons Inc., 2014
Descrizione fisica 1 online resource (499 p.)
Disciplina 572.8/8
Altri autori (Persone) SahuSaura C
Soggetto topico Small interfering RNA
Small interfering RNA - Therapeutic use
Genetic regulation
ISBN 1-118-69603-4
1-118-69599-2
1-118-69601-8
Formato Materiale a stampa
Livello bibliografico Monografia
Lingua di pubblicazione eng
Nota di contenuto pt. I. MicroRNAs and toxicology -- pt. II. MicroRNAs and disease states -- pt. III. MicroRNAs and stem cells -- pt. IV. MicroRNAs and genomics -- pt. V. MicroRNAs and epigenomics -- pt. VI. MicroRNAs and biomarkers.
Record Nr. UNINA-9910139025203321
Chichester, West Sussex, U.K., : John Wiley & Sons Inc., 2014
Materiale a stampa
Lo trovi qui: Univ. Federico II
Opac: Controlla la disponibilità qui
MicroRNAs in toxicology and medicine [[electronic resource] /] / editor, Saura C. Sahu
MicroRNAs in toxicology and medicine [[electronic resource] /] / editor, Saura C. Sahu
Pubbl/distr/stampa Chichester, West Sussex, U.K., : John Wiley & Sons Inc., 2014
Descrizione fisica 1 online resource (499 p.)
Disciplina 572.8/8
Altri autori (Persone) SahuSaura C
Soggetto topico Small interfering RNA
Small interfering RNA - Therapeutic use
Genetic regulation
ISBN 1-118-69603-4
1-118-69599-2
1-118-69601-8
Formato Materiale a stampa
Livello bibliografico Monografia
Lingua di pubblicazione eng
Nota di contenuto pt. I. MicroRNAs and toxicology -- pt. II. MicroRNAs and disease states -- pt. III. MicroRNAs and stem cells -- pt. IV. MicroRNAs and genomics -- pt. V. MicroRNAs and epigenomics -- pt. VI. MicroRNAs and biomarkers.
Record Nr. UNINA-9910816166603321
Chichester, West Sussex, U.K., : John Wiley & Sons Inc., 2014
Materiale a stampa
Lo trovi qui: Univ. Federico II
Opac: Controlla la disponibilità qui