NEW YORK, Dec. 21, 2010 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:
RNAi - technologies, markets and companies
http://www.reportlinker.com/p0203551/RNAi---technologies-markets-and-companies.html
Summary
RNA interference (RNAi) or gene silencing involves the use of double stranded RNA (dsRNA). Once inside the cell, this material is processed into short 21-23 nucleotide RNAs termed siRNAs that are used in a sequence-specific manner to recognize and destroy complementary RNA. The report compares RNAi with other antisense approaches using oligonucleotides, aptamers, ribozymes, peptide nucleic acid and locked nucleic acid.
Various RNAi technologies are described, along with design and methods of manufacture of siRNA reagents. These include chemical synthesis by in vitro transcription and use of plasmid or viral vectors. Other approaches to RNAi include DNA-directed RNAi (ddRNAi) that is used to produce dsRNA inside the cell, which is cleaved into siRNA by the action of Dicer, a specific type of RNAse III. MicroRNAs are derived by processing of short hairpins that can inhibit the mRNAs. Expressed interfering RNA (eiRNA) is used to express dsRNA intracellularly from DNA plasmids.
Delivery of therapeutics to the target tissues is an important consideration. siRNAs can be delivered to cells in culture by electroporation or by transfection using plasmid or viral vectors. In vivo delivery of siRNAs can be carried out by injection into tissues or blood vessels or use of synthetic and viral vectors.
Because of its ability to silence any gene once the sequence is known, RNAi has been adopted as the research tool to discriminate gene function. After the genome of an organism is sequenced, RNAi can be designed to target every gene in the genome and target for specific phenotypes. Several methods of gene expression analysis are available and there is still need for sensitive methods of detection of gene expression as a baseline and measurement after gene silencing. RNAi microarray has been devised and can be tailored to meet the needs for high throughput screens for identifying appropriate RNAi probes. RNAi is an important method for analyzing gene function and identifying new drug targets that uses double-stranded RNA to knock down or silence specific genes. With the advent of vector-mediated siRNA delivery methods it is now possible to make transgenic animals that can silence gene expression stably. These technologies point to the usefulness of RNAi for drug discovery.
RNAi can be rationally designed to block the expression of any target gene, including genes for which traditional small molecule inhibitors cannot be found. Areas of therapeutic applications include virus infections, cancer, genetic disorders and neurological diseases. Side effects can result from unintended interaction between an siRNA compound and an unrelated host gene. If RNAi compounds are designed poorly, there is an increased chance for non-specific interaction with host genes that may cause adverse effects in the host.
Regulatory, safety and patent issues are discussed. There are no major safety concerns and regulations are in preliminary stages as the clinical trials are just starting. Many of the patents are still pending.
The markets for RNAi are difficult to define as no RNAi-based product is approved yet but several are in clinical trials. The major use of RNAi reagents is in research but it partially overlaps that of drug discovery and therapeutic development. Various markets relevant to RNAi are analyzed from 2009 to 2019. Markets are also analyzed according to breakdown of technologies and use of siRNAs, miRNAs, etc.
Profiles of 156 companies involved in developing RNAi technologies are presented along with 204 collaborations. They are a mix of companies that supply reagents and technologies (nearly half of all) and companies that use the technologies for drug discovery. Out of these, 30 are developing RNAi-based therapeutics and 25 are involved in microRNAs. The bibliography contains selected 500 publications that are cited in the report. The text is supplemented with 34 tables and 10 figures.
Table of Contents
0. Executive Summary 15
1. Technologies for suppressing gene function 17
Introduction 17
DNA transcription 17
RNA 17
Non-coding RNA 17
RNA research and potential applications 18
Role of RNA in regulation of the dihydrofolate reductase gene 19
Gene regulation 19
Post-transcriptional regulation of gene expression 20
Alternative RNA splicing 21
Technologies for gene suppression 21
Antisense oligonucleotides 21
Transcription factor decoys 22
Aptamers 22
Ribozymes 23
Aptazymes 23
RNA aptamers vs allosteric ribozymes 23
RNA Lasso 24
Peptide nucleic acid 24
PNA-DNA chimeras 25
Locked nucleic acid 25
Gene silencing 25
Post-transcriptional gene silencing 26
TargeTronO technology for gene knockout 26
Definitions and terminology of RNAi 26
RNAi mechanisms 27
Non-promoter-associated small RNAs 29
Piwi-interacting RNAs in germ cell development 30
Relation of RNAi to junk DNA 30
RNA editing and RNAi 31
Historical landmarks in the development of RNAi 31
2. RNAi Technologies 33
Introduction 33
Comparison of antisense and RNAi 33
Advantages of antisense over siRNAs 33
Advantages of siRNAs over antisense 34
RNA aptamers vs siRNA 34
RNA Lassos versus siRNA 34
Concluding remarks on antisense vs RNAi 35
ssRNAi 35
Antisense vs DNP-ssRNA and DNP-siRNA 35
LNA and RNAi 36
LNA for gene suppression 36
Comparison of LNA and RNAi 37
Use of siLNA to improve siRNA 37
RNAi versus small molecules 37
RNAi in vivo 37
Cre-regulated RNAi in vivo 38
RNAi kits 38
ShortCut™ RNAi Kit 38
HiScribe™ RNAi Transcription Kit 39
pSUPER RNAi system 39
Si2 Silencing Duplex 40
Techniques for measuring RNAi-induced gene silencing 40
Application of PCR in RNAi 40
Real-time quantitative PCR 41
Assessment of the silencing effect of siRNA by RT-PCR 41
Fluorescence resonance energy transfer probe for RNA interactions 42
Bioinformatics tools for design of siRNAs 42
Random siRNA design 42
Rational siRNA design 43
The concept of pooling siRNAs 44
Criteria for rational siRNA design 44
BLOCK-iT RNAi Designer 44
QIAGEN's 2-for-Silencing siRNA Duplexes 45
Designing vector-based siRNA 45
iRNAChek for designing siRNA 45
TROD: T7 RNAi Oligo Designer 45
siDirect: siRNA design software 46
Prediction of efficacy of siRNAs 46
Algorithms for prediction of siRNA efficacy 46
siRNA databases 46
Production of siRNAs 47
Chemical synthesis of short oligonucleotides 47
In vitro transcription 47
Generation of siRNAs in vivo 48
UsiRNAs 48
siRNA:DNA hybrid molecules 49
Chemical modifications of siRNAs 49
Sugar modifications of siRNA 49
Phosphate linkage modifications of siRNA 49
Modifications to the siRNA overhangs 50
Modifications to the duplex architecture 50
Applications of chemical modification of siRNAs 50
Synthetic RNAs vs siRNAs 51
Specificity of siRNAs 51
Asymmetric interfering RNA 52
Genome-wide data sets for the production of esiRNAs 52
ddRNAi for inducing RNAi 52
ddRNAi technology 52
Advantages of ddRNAi over siRNA 53
Short hairpin RNAs 54
siRNA versus shRNA 54
Circular interfering RNA 55
Expressed interfering RNA 56
RNA-induced transcriptional silencing complex 56
Inhibition of gene expression by antigene RNA 57
RNAi vs mRNA modulation by small molecular weight compounds 57
3. MicroRNA 59
Introduction 59
miRNA and RISC 61
Role of the microprocessor complex in miRNA 61
miRNAs compared to siRNAs 62
miRNA and stem cells 63
Influence of miRNA on stem cell formation and maintenance 63
Role of miRNAs in gene regulation during stem cell differentiation 63
miRNA databases 64
Sanger miRBase miRNA sequence database 64
Mapping miRNA genes 64
A database of ultraconserved sequences and miRNA function 65
A database for miRNA deregulation in human disease 65
An database of miRNA-target interactions 66
Role of miRNA in gene regulation 66
Control of gene expression by miRNA 67
miRNA-mediated translational repression involving Piwi 67
Transcriptional regulators of ESCs control of miRNA gene expression 67
Mechanism of miRNAs-induced silencing of gene expression 67
miRNA diagnostics 68
Biochemical approach to identification of miRNA 68
Computational approaches for the identification of miRNAs 69
LNA probes for exploring miRNA 69
Microarrays for analysis of miRNA gene expression 69
Microarrays vs quantitative PCR for measuring miRNAs 70
miRNAs as biomarkers of hepatotoxicity 70
Modification of in situ hybridization for detection of miRNAs 71
Nuclease Protection Assay to measure miRNA expression 71
Real-time PCR for expression profiling of miRNAs 71
Targeting of miRNAs with antisense oligonucleotides 72
Silencing miRNAs by antagomirs 72
New tools for miRNA silencing 72
miRNA-regulated lentiviral vectors 73
miRNAs as drug targets 73
miRNAs as targets for antisense drugs 73
Challenges facing use of miRNAs as drug targets 74
Target specificity of miRNAs 74
Prediction of miRNA targets 75
Role of miRNA in human health and disease 75
Role of miRNAs in regulation of hematopoiesis 75
Role of miRNA depletion in tissue regeneration 76
Role of miRNA in regulation of aging 76
Role of miRNA in inflammation 77
Role of miRNAs in regulation of immune system 77
Role of miRNA in the cardiovascular system 77
Role of miRNAs in development of the cardiovascular system 78
Role of miRNAs in angiogenesis 78
Role of miRNAs in cardiac hypertrophy and failure 78
Role of miRNAs in conduction and rhythm disorders of the heart 79
miRNA-based approach for reduction of hypercholesterolemia 79
miRNA-based approach for restenosis following angioplasty 79
miRNAs as therapeutic targets for cardiovascular diseases 79
Concluding remarks and future prospects of miRNA in the cardiovascular system 80
Role of miRNAs in the nervous system 80
miRNAs and addiction 80
miRNAs in neurodegenerative disorders 81
miRNAs as biomarkers of Alzheimer's disease 81
miRNA malfunction in spinal motor neuron disease 82
miRNAs and retinal neurodegenerative disorders 82
miRNA and schizophrenia 82
Role of miRNA in viral infections 82
Role of miRNA in HSV-1 latency 83
miRNA and autoimmune disorders 83
miRNA in systemic lupus erythematosus 83
miRNA and skin disorders 84
Role of miRNA in inflammatory skin disorders 84
Role of miRNAs in cancer 84
miRNAs linked to the initiation and progression of cancer 84
Oncomirs 84
Linking miRNA sequences to cancer using RNA samples 85
Role of miRNAs in viral oncogenesis 85
miRNA genes in cancer 86
miRNAs, embryonic stem cells and cancer 87
miRNAs and cancer metastases 87
Role of miRNAs in cancer diagnosis 88
Cancer miRNA signature 88
miRNA biomarkers in cancer 88
Diagnostic value of miRNA in cancer 89
Prognostic value of miRNA in cancer 89
miRNAs as basis of cancer therapeutics 89
Antisense oligonucleotides targeted to miRNA 90
Delivery of miRNA mimetics in Cancer 90
Role of miRNAs in adoptive immunotherapy of cancer 90
Restoration of tumor suppressor miRNA may inhibit cancer 91
Role of miRNAs in various cancers 91
miRNA and brain cancer 91
miRNA and breast cancer 92
miRNA and colorectal cancer 92
miRNA and hematological malignancies 93
miRNA and hepatocellular carcinoma 94
miRNA and lung cancer 94
miRNA and nasopharyngeal carcinoma 95
miRNA and ovarian cancer 96
miRNA and pancreatic cancer 96
miRNA and prostatic cancer 97
miRNA and thyroid cancer 97
Future prospects of miRNA 98
Companies involved in miRNA 98
4. Methods of delivery in RNAi 101
Introduction 101
Methods of delivery of oligonucleotides 101
Oral and rectal administration 102
Pulmonary administration 102
Targeted delivery to the CNS 102
High flow microinfusion into the brain parenchyma 103
Intracellular guidance by special techniques 103
Biochemical microinjection 104
Liposomes-mediated oligonucleotide delivery 104
Polyethylenimine-mediated oligonucleotide delivery 104
Delivery of TF Decoys 104
Biodegradable microparticles 105
Microparticles 105
Nanoparticles 105
siRNA delivery technologies 105
Local delivery of siRNA 106
In vivo delivery of siRNAs by synthetic vectors 107
Intracellular delivery of siRNAs 107
Protamine-antibody fusion proteins for delivery of siRNA to cells 107
Protein transduction domains 108
MPG-based delivery of siRNA 108
Delivery of siRNAs with aptamer-siRNA chimeras 108
Phosphorothioate stimulated cellular delivery of siRNA 109
Targeted delivery of siRNAs by lipid-based technologies 109
Delivery of siRNA-lipoplexes 109
Lipidoids for delivery of siRNAs 110
NeoLipid™ technology 110
siFECTamineO 110
Systemic in vivo delivery of lipophilic siRNAs 111
Systemic delivery of siRNAi by lipid nanoparticles 111
Electroporation 111
Nucleofactor technology 112
Intravascular delivery of siRNA 112
27mer siRNA duplexes for improved delivery and potency 113
TransIT-TKOO 113
DNA-based plasmids for delivery of siRNA 114
Convergent transcription 115
PCR cassettes expressing siRNAs 115
Genetically engineered bacteria for delivery of shRNA 115
Viral vectors for delivery of siRNA 115
Adenoviral vectors 116
Adeno-associated virus vectors for shRNA expression 116
Baculovirus vector 116
Lentiviral vectors 117
Retroviral delivery of siRNA 118
Transkingdom RNAi delivery by genetically engineered bacteria 118
Delivery of siRNA without a vector 118
Cell-penetrating peptides for delivery of siRNAs 119
Role of nanobiotechnology in siRNA delivery 119
Chitosan-coated nanoparticles for siRNA delivery 119
Delivery of gold nanorod-siRNA nanoplex to dopaminergic neurons 120
Lipidic aminoglycoside as siRNA nanocarrier 120
Lipid nanoparticles-mediated siRNA delivery 120
Nanosize liposomes for delivery of siRNA 121
PAMAM dendrimers for siRNA delivery 121
Polyethylenimine nanoparticles for siRNA delivery 121
Polycation-based nanoparticles for siRNA delivery 122
Quantum dots to monitor siRNA delivery 122
Targeted delivery of siRNAs to specific organs 123
siRNA delivery to the CNS 123
siRNA delivery to the liver 124
siRNAdelivery to the lungs 124
Control of RNAi and siRNA levels 124
siRNA pharmacokinetics in mammalian cells 125
Mathematical modeling for determining the dosing schedule of siRNA 125
Assessing siRNA pharmacodynamics in animal models 126
Research on siRNA delivery funded by the NIH 126
Companies involved in delivery technologies for siRNA 127
5. RNAi in Research 131
Introduction 131
Basic RNAi research 131
Genes and lifespan 131
Antiviral role of RNAi in animal cells 131
Silencing snoRNA genes 131
Profiling small RNAs 132
Study of signaling pathways 132
RNAi for research in neuroscience 132
Use of RNAi to study insulin action 133
Detection of cancer mutations 133
Loss-of-function genetic screens 133
Inducible and reversible RNAi 134
Combination of siRNA with green fluorescent protein 134
RNAi and environmental research 134
Applied RNAi research 135
RNAi for gene expression studies 135
Microarrays for measuring gene expression in RNAi 135
RNAi for functional genomic analysis 136
RNAi studies on C. elegans 136
RNAi studies on Drosophila 137
RNAi in planaria 137
Testing the specificity of RNAi 138
Tissue-specific RNAi 138
siRNA-mediated gene silencing 138
RNAi libraries 139
Universal plasmid siRNA library 140
pDual library using plasmid vector 140
pHippy plasmid vector library 140
siRNA libary including miRNAs 140
siRNA libraries using pRetroSuper vector 141
siRNA produced by enzymatic engineering of DNA 141
shRNA libraries 141
Enzymatic production of RNAi library 142
RNAi and alternative splicing 143
RNAi in animal development 143
RNAi for creating transgenic animals 143
RNAi for creating models of neurological disorders 144
Research support for RNAi in US 144
RNAi for toxicogenomics 144
Role of RNAi in the US biodefense research 145
The RNAi Consortium 145
Research support for RNAi in Europe 146
European Union for RNA Interference Technology 146
Research support of RNAi 146
Role of RNAi in MitoCheck project 147
RNAi Global Initiative 147
6. RNAi in drug discovery 151
Basis of RNAi for drug discovery 151
Use of siRNA libraries to identify genes as therapeutic targets 151
Role of siRNAs in drug target identification 151
Use of a genome-wide, siRNA library for drug discovery 152
Use of arrayed adenoviral siRNA libraries for drug discovery 152
RNAi as a tool for assay development 152
Targeting human kinases with an siRNAi library 153
Challenges of drug discovery with RNAi 153
Express TrackSM siRNA Drug Discovery Program 153
Genome-wide siRNA screens in mammalian cells 154
Natural antisense and ncRNA as drug targets 154
RNAi for target validation 155
Delivering siRNA for target validation in vivo 155
Off-target effects of siRNA-mediated gene silencing 157
Bioinformatic approach to off-target effects 158
Validation of oncology targets discovered through RNAi screens 158
Selection of siRNA versus shRNA for target validation 158
Application of RNAi to the druggable genome 159
Application of siRNA during preclinical drug development 159
siRNAs vs small molecules as drugs 160
siRNAs vs antisense drugs 160
RNAi technology in plants for drug discovery and development 161
Application of RNAi to poppy plant as source of new drugs 161
7. Therapeutic applications of RNAi 163
Introduction 163
Potential of RNAi-based therapies 164
In vitro applications of siRNA 164
In vivo applications of RNAi 165
RNAi and cell therapy 165
Gene inactivation to study hESCs 166
RNAi and stem cells 166
Cell therapy for immune disorders 167
RNAi gene therapy 167
Drug-inducible systems for control of gene expression 167
Potential side effects of RNAi gene therapy 168
Systemic delivery of siRNAs 168
In vivo RNAi therapeutic efficacy in animal models of human diseases 169
Virus infections 169
RNAi approaches to viral infections 170
Delivery of siRNAs in viral infections 171
RNAi applications in HIV 171
A multiple shRNA approach for silencing of HIV-1 172
Anti-HIV shRNA for AIDS lymphoma 172
Aptamer-mediated delivery of anti-HIV siRNAs 172
Bispecific siRNA constructs 172
Role of the nef gene during HIV-1 infection and RNAi 173
siRNA-directed inhibition of HIV-1 infection 173
Synergistic effect of snRNA and siRNA 174
Targeting CXCR4 with siRNAs 174
Targeting CCR5 with siRNAs 174
Concluding remarks on RNAi approach to HIV/AIDS 175
Influenza 175
Inhibition of influenza virus by siRNAs 176
Delivery of siRNA in influenza 177
Challenges and future prospects of siRNAs for influenza 177
Respiratory syncytial and parainfluenza viruses 178
Coronavirus/severe acute respiratory syndrome 179
Herpes simplex virus 2 179
Hepatitis B 179
Hepatitis C virus 180
Cytomegalovirus 181
siRNA vs antisense oligonucleotides for viral infections 182
siRNA against methicillin-resistant S. aureus 182
RNAi-based rational approach to antimalarial drug discovery 183
Inhibiting the growth of malarial parasite by heme-binding DNA aptamers 183
siRNA-based antimalarial therapeutics 183
RNAi applications in oncology 184
Inhibition of oncogenes 184
RNAi approach to study TRAIL 186
Modification of alternative splicing in cancer 186
Allele-specific inhibition 186
siRNAs for anticancer drug discovery 187
siRNAs for inducing cancer immunity 188
siRNAs for inhibition of angiogenesis 188
siRNA targeting the R2 subunit of ribonucleotide reductase 189
siRNA for cancer chemoprevention 189
Onconase 189
Drug delivery issues in managing cancer by RNAi approach 190
siHybrids vs siRNAs as anticancer agents 190
Nanobiotechnology-based delivery of siRNAs 191
Lipid nanoparticle-based delivery of anticancer siRNAs 191
Minicells for targeted delivery of nanoscale anticancer therapeutics 191
Nanoimmunoliposome-based system for targeted delivery of siRNA 192
Polymer nanoparticles for targeted delivery of anticancer siRNA 192
RNA nanotechnology for delivery of cancer therapeutics 193
Targeted delivery of a nanoparticle-siRNA complex in cancer patients 193
RNAi-based treatment of various cancer types 194
RNAi-based therapy of brain cancer 194
RNAi in breast cancer 196
Enhancing efficacy of hyperthermia/chemotherapy in cervical cancer 196
RNAi and colorectal cancer 196
RNAi and Ewing's sarcoma 197
RNAi and leukemias 197
RNAi and lung cancer 198
RNAi and melanoma 198
RNAi and pancreatic cancer 199
RNAi and prostate cancer 199
Overcoming drug resistance in cancer 200
Targeting fusion proteins in cancer 200
Increasing chemosensitivity by RNAi 200
Genetic disorders 201
RNAi for skin disorders 201
Experimental studies for RNAi applications in skin disorders 201
Clinical applications of RNAi in skin disorders 202
Pachyonychia congenita 202
Neurological disorders 203
RNAi for neurodegenerative disorders 204
Alzheimer's disease 204
Parkinson's disease 205
Amyotrophic lateral sclerosis 205
Prion diseases 206
Polyglutamine-induced neurodegeneration 207
Fragile X syndrome and RNAi 207
RNAi-based therapy for Huntington's disease 208
Combination of RNAi and gene therapy to prevent neurodegenerative disease 209
Role of RNAi in pain therapy 209
Role of RNAi in repair of spinal cord injury 210
Role of RNAi in treatment of multiple sclerosis 210
siRNA for Duchenne muscular dystrophy 211
siRNA for dystonia 211
RNAi in ophthalmology 211
Age related macular degeneration 211
Current treatment of AMD 212
RNAi-based treatments for AMD 213
Diabetic retinopathy 214
Retinitis pigmentosa 214
RNAi and metabolic disorders 215
RNAi and obesity 215
Genes and regulation of body fat 215
RNAi and diabetes 215
Use of siRNAs to study glucose transporter 215
Use of RNAi to study genes in animal models of diabetes 216
RNAi for drug discovery in diabetes 216
A miRNA that regulates insulin secretion 217
RNAi in hematology 218
Stem cell-based gene therapy and RNAi for sickle cell disease 218
RNAi and disorders of the immune system 219
siRNA applications in immunology 219
Use of RNAi in transplantation 220
RNAi for cardiovascular disorders 220
RNAi for hypercholesterolemia 220
siRNA targeting NADPH oxidase in cardiovascular diseases 221
siRNA for study and treatment of ischemia-reperfusion injury 221
RNAi in respiratory disorders 222
siRNA for cystic fibrosis 222
siRNA for asthma 223
RNAi for musculoskeletal disorders 223
RNAi for rheumatoid arthritis 223
RNAi for bone disorders 224
RNAi for treatment of osteoporosis 224
Clinical trials of RNAi-based therapies 225
Improving efficacy of siRNAs for clinical trials by improved delivery 225
Role of RNAi in development of personalized medicine 226
Future prospects of RNAi 226
Challenges for the development of RNAi-based therapeutics 227
8. Safety, regulatory and patent issues 229
Introduction 229
Limitations and drawbacks of RNAi 229
Adverse effects of RNAi 229
Effect of siRNAs on interferon response 230
Detection of interferon response 230
Prevention of the interferon response in RNAi 231
Overcoming the innate immune response to siRNAs 231
Toxicity associated with RNAi 232
Selection of siRNAs to improve specificity and efficacy 232
Regulatory issues relevant to RNAi 232
RNAi patents 233
Companies with strong patent position 233
Alnylam 233
Benitec 236
Intradigm 236
Quark Pharmaceuticals 236
Sirna Therapeutics 237
9. Markets for RNAi Technologies 239
Introduction 239
Current and future market potential for RNAi technologies 239
RNAi reagents 240
RNAi-based drug discovery and target validation 240
RNAi-based development of therapeutics 240
RNAi market potential according to therapeutic areas 240
Market for viral infections 241
Market for cancer 242
Market for age related macular degeneration 242
Unmet needs in RNAi 242
Strategies for marketing RNAi 243
Choosing optimal indications 243
Strategies according to the trends in healthcare in the next decade 244
Concluding remarks 245
10. Companies involved in RNAi Technologies 247
Introduction 247
Major players in RNAi 250
Profiles of companies 251
Collaborations 432
11. References 439
Tables
Table 1-1: Classification of small RNA molecules 27
Table 1-2: Mechanisms of small RNAs involved in gene silencing 28
Table 1-3: Historical landmarks in the evolution of RNAi 31
Table 2-1: RNAi versus small molecules 37
Table 2-2: Providers of software for siRNA design 43
Table 2-3: Methods for the production of siRNAs 47
Table 2-4: Advantages and limitations of methods of shRNA-derived siRNA knockdown 55
Table 2-5: Comparison of eiRNA with siRNA 56
Table 3-1: Methods for miRNA target prediction 75
Table 3-2: miRNA expression in neurodegenerative diseases 81
Table 3-3: Dysregulation of miRNA expression in epithelial cancers 84
Table 3-4: Companies involved in miRNA diagnostics and therapeutics 98
Table 4-1: Methods of delivery of oligonucleotides 101
Table 4-2: Methods of delivery of siRNA 106
Table 4-3: Companies developing siRNA delivery technologies 127
Table 5-1: RNAi libraries 139
Table 6-1: Delivery of siRNAs in vivo for target validation 156
Table 6-2: Selection of siRNA versus shRNA for target validation 159
Table 7-1: RNAi-based therapeutic approaches 164
Table 7-2: In vivo RNAi therapeutic efficacy in animal models of human diseases 169
Table 7-3: Inhibition of viral replication by RNAi 170
Table 7-4: Cancer-associated genes that can be targeted by RNAi 185
Table 7-5: Neurological disorders that have been studied by using RNAi 203
Table 7-6: Clinical trials of RNAi-based therapeutics 225
Table 9-1: RNAi markets according to technologies and reagents 2009-2019 239
Table 9-2: Markets for RNAi therapy for selected diseases: years 2009-2019 241
Table 10-1: RNAi reagent, technology and service companies 247
Table 10-2: Pharmaceutical companies using RNAi for drug discovery and development 248
Table 10-3: Biotechnology companies using RNAi for drug discovery and development 249
Table 10-4: Companies developing RNAi-based therapeutic products 250
Table 10-5: Major players in RNAi 250
Table 10-6: RNAi products of Benitec 270
Table 10-7: Proprietary reagents of ImuThes 322
Table 10-8: Product pipeline of Silence Therapeutics 403
Table 10-9: Collaborations in RNAi technologies 432
Figures
Figure 1-1: Relationship of DNA, RNA and protein in the cell 20
Figure 1-2: Schematic of suppression of gene expression by RNAi 28
Figure 2-1: Overview of ShortCut RNAi Kit 39
Figure 2-2: Gene silencing by RNAi induced with ddRNAi 53
Figure 3-1: A schematic miRNA pathway 59
Figure 3-2: Molecular mechanisms of miRNA generation 60
Figure 7-1: Targeting disease by RNAi 163
Figure 7-2: Role of RNAi in personalized medicine 226
Figure 8-1: Problems with use of synthetic siRNAs and measures to prevent them 230
Figure 9-1: Unmet needs in RNAi technologies 243
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Pharmaceutical Industry: RNAi - technologies, markets and companies
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Nicolas Bombourg
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