Reportlinker Adds RNAi - technologies, markets and companies

Dec 21, 2010, 12:20 ET from Reportlinker

NEW YORK, Dec. 21, 2010 /PRNewswire/ -- announces that a new market research report is available in its catalogue:

RNAi - technologies, markets and companies


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


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


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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Nicolas Bombourg



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