+353-1-416-8900REST OF WORLD
1-800-526-8630U.S. (TOLL FREE)

RNAi - Technologies, Markets and Companies

  • ID: 42874
  • Report
  • March 2017
  • Region: Global
  • 521 Pages
  • Jain PharmaBiotech
1 of 4

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. Research at academic centers that is relevant to RNAi-based therapeutics is mentioned.

Regulatory, safety and patent issues are discussed. 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. However, there are no major safety concerns and regulations are in preliminary stages as the clinical trials are still ongoing and there are no marketed products. 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 2016 to 2026. Markets are also analyzed according to technologies and use of siRNAs, miRNAs, etc.

Profiles of 162 companies involved in developing RNAi technologies are presented along with 235 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, 33 are developing RNAi-based therapeutics and 36 are involved in microRNAs. The bibliography contains selected 650 publications that are cited in the report. The text is supplemented with 38 tables and 15 figures.

Note: Product cover images may vary from those shown
2 of 4

0. Executive Summary

1. Technologies for suppressing gene function
DNA transcription
Non-coding RNA
RNA research and potential applications
Role of RNA in regulation of the dihydrofolate reductase gene
Gene regulation
Post-transcriptional regulation of gene expression
Alternative RNA splicing
Technologies for gene suppression
Antisense oligonucleotides
Phosphorodiamidate morpholino oligomers
Transcription factor decoys
RNA aptamers vs allosteric ribozymes
Peptide nucleic acid
PNA-DNA chimeras
Locked nucleic acid
Long noncoding RNAs
Gene silencing
Post-transcriptional gene silencing
TargeTron? technology for gene knockout
Definitions and terminology of RNAi
RNAi mechanisms
RNAi oligonucleotides
Non-promoter-associated small RNAs
Piwi-interacting RNAs in germ cell development
Relation of RNAi to junk DNA
RNAi and epigenetic mechanisms
RNA editing and RNAi
Historical landmarks in the development of RNAi

2. RNAi Technologies
Comparison of antisense and RNAi
Advantages of antisense over siRNAs
Advantages of siRNAs over antisense
RNA aptamers vs siRNA
RNA Lassos versus siRNA
Concluding remarks on antisense vs RNAi
PMOs vs antisense and siRNAs
Antisense vs DNP-ssRNA and DNP-siRNA
LNA and RNAi
LNA for gene suppression
Comparison of LNA and RNAi
Use of siLNA to improve siRNA
RNAi versus small molecules
RNAi in vivo
Cre-regulated RNAi in vivo
RNAi kits
ShortCut™ RNAi Kit
HiScribe™ RNAi Transcription Kit
pSUPER RNAi system
Si2 Silencing Duplex
Techniques for measuring RNAi-induced gene silencing
Application of PCR in RNAi
Real-time quantitative PCR
Assessment of the silencing effect of siRNA by RT-PCR
Fluorescence resonance energy transfer probe for RNA interactions
Bioinformatics tools for design of siRNAs
Random siRNA design
Rational siRNA design
The concept of pooling siRNAs
Criteria for rational siRNA design
BLOCK-iT RNAi Designer
QIAGEN's 2-for-Silencing siRNA Duplexes
Designing vector-based siRNA
iRNAChek for designing siRNA
TROD: T7 RNAi Oligo Designer
siDirect: siRNA design software
Prediction of efficacy of siRNAs
Algorithms for prediction of siRNA efficacy
siRNA databases
Production of siRNAs
Chemical synthesis of short oligonucleotides
In vitro transcription
Generation of siRNAs in vivo
siRNA:DNA hybrid molecules
Chemical modifications of siRNAs
Sugar modifications of siRNA
Phosphate linkage modifications of siRNA
Modifications to the siRNA overhangs
Modifications to the duplex architecture
Applications of chemical modification of siRNAs
Synthetic RNAs vs siRNAs
Specificity of siRNAs
Asymmetric interfering RNA
Genome-wide data sets for the production of esiRNAs
ddRNAi for inducing RNAi
ddRNAi technology
Advantages of ddRNAi over siRNA
Short hairpin RNAs
siRNA versus shRNA
Circular interfering RNA
Expressed interfering RNA
RNA-induced transcriptional silencing complex
Inhibition of gene expression by antigene RNA
RNAi vs mRNA modulation by small molecular weight compounds
saRNA for transcriptional activation

3. MicroRNA
Circular RNA and miRNA
miRNA and RISC
Role of the microprocessor complex in miRNA
miRNAs compared to siRNAs
miRNA and stem cells
Influence of miRNA on stem cell formation and maintenance
Role of miRNAs in gene regulation during stem cell differentiation
miRNA databases
Sanger miRBase miRNA sequence database
Mapping miRNA genes
A database of ultraconserved sequences and miRNA function
A database for miRNA deregulation in human disease
An database of miRNA-target interactions
Role of miRNA in gene regulation
Control of gene expression by miRNA
miRNA-mediated translational repression involving Piwi
Transcriptional regulators of ESCs control of miRNA gene expression
Mechanism of miRNAs-induced silencing of gene expression
miRNA diagnostics
Biochemical approach to identification of miRNA
Computational approaches for the identification of miRNAs
LNA probes for exploring miRNA
miR-TRAP to identify miRNA targets in vivo
Microarrays for analysis of miRNA gene expression
Microarrays vs quantitative PCR for measuring miRNAs
miRNAs as biomarkers of hepatotoxicity
Modification of in situ hybridization for detection of miRNAs
Nuclease Protection Assay to measure miRNA expression
Real-time PCR for expression profiling of miRNAs
Significance of miRNAs in diagnostics
Targeting of miRNAs with antisense oligonucleotides
Silencing miRNAs by antagomirs
New tools for miRNA silencing
Use of HAPIscreen for identification of aptamers against pre-miRNAs
miRNA-regulated lentiviral vectors
miRNAs as drug targets
miRNAs as targets for antisense drugs
Challenges facing use of miRNAs as drug targets
Target specificity of miRNAs
Prediction of miRNA targets
Role of miRNA in human health and disease
Role of miRNAs in regulation of hematopoiesis
Role of miRNA depletion in tissue regeneration
Role of miRNA in regulation of aging
Role of miRNA in inflammation
Role of miRNAs in regulation of immune system
Role of miRNA in diabetes
Role of miRNA in the cardiovascular system
Role of miRNAs in development of the cardiovascular system
Role of miRNAs in angiogenesis
Role of miRNA in atherosclerosis
Role of miRNAs in cardiac hypertrophy and failure
Role of miRNA in cardiomyopathy
Role of miRNAs in conduction and rhythm disorders of the heart
Diagnostic and prognostic value of miRNAs in acute coronary syndrome
miRNA-based approaches for reduction of hypercholesterolemia
miRNA-based approach for restenosis following angioplasty
miRNA gene therapy for ischemic heart disease
miRNAs as therapeutic targets for cardiovascular diseases
Concluding remarks and future prospects of miRNA in the cardiovascular system
Role of miRNAs in diseases of the eye
Pathological ocular neovascularization
Role of miRNAs in diabetic retinopathy
Role of miRNAs in glaucoma
Role of miRNA in corneal scarring
Role of miRNAs in the nervous system
miRNAs and addiction
miRNAs in neurodegenerative disorders
miRNAs as biomarkers of Alzheimer’s disease
miRNAs in Parkinson disease
miRNAs in Huntington’s disease
miRNAs in ALS
miRNAs as biomarkers of prion-induced neurodegeneration
miRNAs and retinal neurodegenerative disorders
miRNA and stroke
miRNA and schizophrenia
Role of miRNA in neuroprotection
Role of miRNA in viral infections
Role of miRNA in HSV-1 latency
miRNA and autoimmune disorders
miRNA in rheumatoid arthritis
miRNA in systemic lupus erythematosus
miRNAs in gastrointestinal disorders
miRNA-based therapies for the irritable bowel syndrome
miRNA and skin disorders
Role of miRNA in inflammatory skin disorders
Role of miRNA in hypertrophic scarring of the skin
Role of miRNAs in cancer
miRNAs linked to the initiation and progression of cancer
Linking miRNA sequences to cancer using RNA samples
Role of miRNAs in viral oncogenesis
miRNA genes in cancer
miRNAs interaction with p53
miRNAs, embryonic stem cells and cancer
miRNAs and cancer metastases
Role of miRNAs in cancer diagnosis
Cancer miRNA signature
miRNA biomarkers in cancer
Diagnostic value of miRNA in cancer
Prognostic value of miRNA in cancer
miRNA-based cancer therapeutics
Antisense oligonucleotides targeted to miRNA
Role of miRNAs in adoptive immunotherapy of cancer
Restoration of tumor suppressor miRNAs to inhibit cancer
Delivery strategies for miRNA modulators in cancer
Role of miRNAs in various cancers
miRNA and malignant gliomas of the brain
miRNAs and CNS tumors in children
miRNAs and pituitary tumors
miRNA and breast cancer
miRNA and colorectal cancer
miRNA and gastrointestinal cancer
miRNA and leukemia
miRNA and lymphomas
miRNA and hepatocellular carcinoma
miRNA and lung cancer
miRNA and nasopharyngeal carcinoma
miRNA and ovarian cancer
miRNA and pancreatic cancer
miRNA and prostatic cancer
miRNA and thyroid cancer
Companies involved in miRNA
Status of therapeutic development of miRNAs
Concluding remarks and future prospects of miRNA therapeutics

4. Methods of delivery in RNAi
Methods of delivery of oligonucleotides
Oral and rectal administration
Pulmonary administration
Targeted delivery to the CNS
High flow microinfusion into the brain parenchyma
Intracellular guidance by special techniques
Biochemical microinjection
Liposomes-mediated oligonucleotide delivery
Polyethylenimine-mediated oligonucleotide delivery
Delivery of TF Decoys
Biodegradable microparticles
Self-delivering rxRNA
siRNA delivery technologies
Local delivery of siRNA
In vivo delivery of siRNAs by synthetic vectors
Intracellular delivery of siRNAs
Delivery of siRNAs with aptamer-siRNA chimeras
MPG-based delivery of siRNA
Protamine-antibody fusion proteins for delivery of siRNA to cells
Protein transduction domains
Phosphorothioate stimulated cellular delivery of siRNA
Targeted delivery of siRNAs by lipid-based technologies
Delivery of siRNA-lipoplexes
Lipidoids for delivery of siRNAs
NeoLipid™ technology
Systemic in vivo delivery of lipophilic siRNAs
Challenges and future prospects of lipid-based siRNA delivery
Nucleofactor technology
Visualization of electrotransfer of siRNA at single cell level
Intravascular delivery of siRNA
27mer siRNA duplexes for improved delivery and potency
DNA-based plasmids for delivery of siRNA
Convergent transcription
PCR cassettes expressing siRNAs
Genetically engineered bacteria for delivery of shRNA
Viral vectors for delivery of siRNA
Adenoviral vectors
Adeno-associated virus vectors for shRNA expression
Baculovirus vector
Lentiviral vectors
Retroviral delivery of siRNA
Transkingdom RNAi delivery by genetically engineered bacteria
Delivery of siRNA without a vector
Cell-penetrating peptides for delivery of siRNAs
Role of nanobiotechnology in siRNA delivery
Chitosan-coated nanoparticles for siRNA delivery
Cyclodextrin nanoparticles
Delivery of gold nanorod-siRNA nanoplex to dopaminergic neurons
Lipidic aminoglycoside as siRNA nanocarrier
Lipid nanoparticles-mediated siRNA delivery
Nanoparticles for intracellular delivery of siRNA
Nanosize liposomes for delivery of siRNA
PAMAM dendrimers for siRNA delivery
PEG-PCL-DEX polymersome-protamine vector
Polyethylenimine nanoparticles for siRNA delivery
Polycation-based nanoparticles for siRNA delivery
Quantum dots to monitor siRNA delivery
siRNA-nanoparticle conjugates for improving stability in serum
Systemic delivery of siRNAi by lipid nanoparticles
Topical delivery of siRNA-nanoparticle conjugates
Targeted delivery of siRNAs to specific organs
siRNA delivery to the CNS
siRNA delivery to the liver
siRNA delivery to the lungs
Control of RNAi and siRNA levels
siRNA pharmacokinetics in mammalian cells
Mathematical modeling for determining the dosing schedule of siRNA
Assessing siRNA pharmacodynamics in animal models
Research on siRNA delivery funded by the NIH
Companies involved in delivery technologies for siRNA

5. RNAi in Research
Basic RNAi research
Antiviral role of RNAi in animal cells
Combination of siRNA with green fluorescent protein
Detection of cancer mutations
Genes and lifespan
Inducible and reversible RNAi
Loss-of-function genetic screens
Nanoparticles mimic RNAi
Profiling small RNAs
RNAi for research in neuroscience
RNAi and environmental research
Small nucleolar RNAs
Study of signaling pathways
Transgenic RNAi
Use of RNAi to study insulin action
Applied RNAi research
RNAi for gene expression studies
Microarrays for measuring gene expression in RNAi
RNAi for functional genomic analysis
RNAi studies on C. elegans
RNAi studies on Drosophila
RNAi in planaria
RNAi for regenerative medicine
Testing the specificity of RNAi
Tissue-specific RNAi
siRNA-mediated gene silencing
RNAi libraries
Enzymatic production of RNAi library
Next-generation libraries for RNAi-based genome-wide screens
pDual library using plasmid vector
pHippy plasmid vector library
siRNA libraries using pRetroSuper vector
siRNA produced by enzymatic engineering of DNA
shRNA libraries
RNAi and alternative splicing
RNAi in animal development
RNAi for creating transgenic animals
RNAi for creating models of neurological disorders
Research support for RNAi in US
RNAi for toxicogenomics
Role of RNAi in the US biodefense research
The RNAi Consortium
Research support for RNAi in Europe
European Union for RNA Interference Technology
Research support of RNAi
Role of RNAi in MitoCheck project
RNAi Global Initiative
SIROCCO project

6. RNAi in drug discovery
Basis of RNAi for drug discovery
RIP-Chip for study of RNA-protein interactions
RNAi for identification of genes as therapeutic targets
Role of siRNAs in drug target identification
Use of a genome-wide, siRNA library for drug discovery
Use of arrayed adenoviral siRNA libraries for drug discovery
RNAi as a tool for assay development
Targeting human kinases with an siRNAi library
Challenges of drug discovery with RNAi
Express Track
SM siRNA Drug Discovery Program
Genome-wide siRNA screens in mammalian cells
Natural antisense and ncRNA as drug targets
RNAi for target validation
Delivering siRNA for target validation in vivo
Validation of oncology targets discovered through RNAi screens
Selection of siRNA versus shRNA for target validation
Off-target effects of siRNA-mediated gene silencing
Bioinformatic approach to off-target effects
siPools for eliminating off-target effects of siRNAs
Managing off-target effects of vector-encoded shRNAs
Application of RNAi to the druggable genome
Application of siRNA during preclinical drug development
siRNAs vs small molecules as drugs
siRNAs vs antisense drugs
Chemical modifications for improving siRNA drugs
RNAi technology in plants for drug discovery and development
Application of RNAi to poppy plant as source of new drugs

7. Therapeutic applications of RNAi
Potential of RNAi-based therapies
In vitro applications of siRNA
In vivo applications of RNAi
RNAi and cell therapy
Gene inactivation to study hESCs
RNAi and stem cells
RNAi for study of ESCs
RNAi and iPSCs
Cell therapy for immune disorders
RNAi gene therapy
Drug-inducible systems for control of gene expression
Potential side effects of RNAi gene therapy
Systemic delivery of siRNAs
In vivo RNAi therapeutic efficacy in animal models of human diseases
Role of RNAi in regenerative medicine
Virus infections
RNAi approaches to viral infections
Delivery of siRNAs in viral infections
RNAi applications in HIV
A multiple shRNA approach for silencing of HIV-1
Anti-HIV shRNA for AIDS lymphoma
Aptamer-mediated delivery of anti-HIV siRNAs
Bispecific siRNA constructs
Role of the nef gene during HIV-1 infection and RNAi
siRNA-directed inhibition of HIV-1 infection
Synergistic effect of snRNA and siRNA
Targeting CXCR4 with siRNAs
Targeting CCR5 with siRNAs
Concluding remarks on RNAi approach to HIV/AIDS
Inhibition of influenza virus by siRNAs
Delivery of siRNA in influenza
Challenges and future prospects of siRNAs for influenza
Respiratory syncytial and parainfluenza viruses
Coronavirus/severe acute respiratory syndrome
Herpes simplex virus 2
Hepatitis B
Hepatitis C virus
Virus causing hemorrhagic fever
Dengue fever
Antivirals in development
Ebola virus
Marburg virus
siRNA vs antisense oligonucleotides for viral infections
siRNA against methicillin-resistant S. aureus
RNAi-based rational approach to antimalarial drug discovery
Inhibiting the growth of malarial parasite by heme-binding DNA aptamers
siRNA-based antimalarial therapeutics
RNAi applications in oncology
Allele-specific inhibition
Drug delivery issues in managing cancer by RNAi approach
Inhibition of oncogenes
Modification of alternative splicing in cancer
Overcoming drug resistance in cancer
Targeting fusion proteins in cancer
Increasing chemosensitivity by RNAi
RNAi approach to study TRAIL
RNAi-based logic circuit for identification of specific cancer cells
shRNA-based autologous cancer vaccine
siRNAs for anticancer drug discovery
siRNAs for inducing cancer immunity
siRNAs for inhibition of angiogenesis
siRNA targeting the R2 subunit of ribonucleotide reductase
siRNA for cancer chemoprevention
siHybrids vs siRNAs as anticancer agents
Nanobiotechnology-based delivery of siRNAs
Lipid nanoparticle-based delivery of anticancer siRNAs
Minicells for targeted delivery of nanoscale anticancer therapeutics
Nanoimmunoliposome-based system for targeted delivery of siRNA
PEG-nanoparticles for delivery of siRNA to target fusion genes
Polymer nanoparticles for targeted delivery of anticancer siRNA
RNA nanotechnology for delivery of cancer therapeutics
siRNA delivery in combination with nanochemotherapy
Targeted delivery of a nanoparticle-siRNA complex in cancer patients
RNAi-based treatment of various cancer types
RNAi-based therapy of glioblastoma multiforme
RNAi in breast cancer
RNAi for enhancing hyperthermia/chemotherapy in cervical cancer
RNAi and colorectal cancer
RNAi and Ewing’s sarcoma
RNAi and leukemias
RNAi and lung cancer
RNAi and melanoma
RNAi and pancreatic cancer
RNAi and prostate cancer
Genetic disorders
RNAi for skin disorders
Experimental studies for RNAi applications in skin disorders
Clinical applications of RNAi in skin disorders
Pachyonychia congenita
Skin scarring
Neurological disorders
RNAi for neurodegenerative disorders
Alzheimer's disease
Parkinson's disease
Amyotrophic lateral sclerosis
Prion diseases
Polyglutamine-induced neurodegeneration
Fragile X syndrome and RNAi
RNAi-based therapy for Huntington's disease
Combination of RNAi and gene therapy to prevent neurodegenerative disease
Role of RNAi in pain therapy
Role of RNAi in repair of spinal cord injury
Role of RNAi in treatment of multiple sclerosis
siRNA for Duchenne muscular dystrophy
siRNA for dystonia
RNAi in ophthalmology
Age related macular degeneration
Current treatment of AMD
RNAi-based treatments for AMD
Diabetic retinopathy
Retinitis pigmentosa
RNAi and metabolic disorders
RNAi and obesity
Genes and regulation of body fat
RNAi and diabetes
Regulation of insulin secretion by a miRNA
RNAi for study of genes in animal models of diabetes
RNAi for drug discovery in diabetes
RNAi for treating liver dysfunction in diabetes
siRNAs for study of glucose transporter
siRNAs for targeting adipose inflammation in diabetes and obesity
RNAi in hematology
Stem cell-based gene therapy and RNAi for sickle cell disease
RNAi and disorders of the immune system
siRNA applications in immunology
Use of RNAi in transplantation
RNAi for cardiovascular disorders
RNAi for hypercholesterolemia
siRNAs targeting PCSK9
siRNA targeting NADPH oxidase in cardiovascular diseases
Role of lncRNA in cardiovascular disorders
siRNA for study and treatment of ischemia-reperfusion injury
RNAi in respiratory disorders
siRNA for cystic fibrosis
siRNA for asthma
RNAi for musculoskeletal disorders
RNAi for rheumatoid arthritis
RNAi for bone disorders
RNAi for treatment of osteoporosis
RNAi for miscellaneous disorders
RNAi for transthyretin amyloidosis
Research relevant to RNAi-based therapies at academic institutes
Laboratory of RNA Molecular Biology, The Rockefeller University
RNAi Center, La Jolla Institute for Allergy & Immunology
Clinical trials of RNAi-based therapies
Improving efficacy of siRNAs for clinical trials by improved delivery
Role of RNAi in development of personalized medicine
Future prospects of RNAi
Challenges for the development of RNAi-based therapeutics

8. Safety, regulatory and patent issues
Limitations and drawbacks of RNAi
Adverse effects of RNAi
Effect of siRNAs on interferon response
Detection of interferon response
Prevention of the interferon response in RNAi
Overcoming the innate immune response to siRNAs
Toxicity associated with RNAi
Selection of siRNAs to improve specificity and efficacy
Regulatory issues relevant to RNAi
RNAi patents
Companies with strong patent position
Quark Pharmaceuticals
Sirna Therapeutics

9. Markets for RNAi Technologies
Current and future market potential for RNAi technologies
RNAi reagents
siRNA markets
miRNA markets
RNAi-based drug discovery and target validation
RNAi-based development of therapeutics
RNAi market potential according to therapeutic areas
Market for viral infections
Market for cancer
Market for age related macular degeneration
Unmet needs in RNAi
Strategies for marketing RNAi
Choosing optimal indications
Strategies according to the trends in healthcare in the next decade
Concluding remarks

10. Companies involved in RNAi Technologies
Major players in RNAi
Profiles of companies

11. References

List of Tables
Table 1-1: Classification of small RNA molecules
Table 1-2: Mechanisms of small RNAs involved in gene silencing
Table 1-3: Historical landmarks in the evolution of RNAi
Table 2-1: RNAi versus small molecules
Table 2-2: Providers of software for siRNA design
Table 2-3: Methods for the production of siRNAs
Table 2-4: Advantages and limitations of methods of shRNA-derived siRNA knockdown
Table 2-5: Comparison of eiRNA with siRNA
Table 3-1: Methods for miRNA target prediction
Table 3-2: miRNA expression in neurodegenerative diseases
Table 3-3: Dysregulation of miRNA expression in epithelial cancers
Table 3-4: Delivery strategies for miRNA inhibition therapies in cancer
Table 3-5: Delivery strategies for miRNA replacement to inhibit cancer growth
Table 3-6: MiRNAs and key molecules in signaling pathways in glioblastoma multiforme
Table 3-7: Companies involved in miRNA diagnostics and therapeutics
Table 3-8: Status of therapeutic development of miRNAs
Table 4-1: Methods of delivery of oligonucleotides
Table 4-2: Methods of delivery of siRNA
Table 4-3: Companies developing siRNA delivery technologies
Table 5-1: RNAi libraries
Table 6-1: Delivery of siRNAs in vivo for target validation
Table 6-2: Selection of siRNA versus shRNA for target validation
Table 7-1: RNAi-based therapeutic approaches
Table 7-2: In vivo RNAi therapeutic efficacy in animal models of human diseases
Table 7-3: Inhibition of viral replication by RNAi
Table 7-4: Cancer-associated genes that can be targeted by RNAi
Table 7-5: Neurological disorders that have been studied by using RNAi
Table 7-6: Clinical trials of RNAi-based therapeutics
Table 9-1: RNAi markets according to technologies and reagents 2016-2026
Table 9-2: Markets for RNAi therapy for selected diseases: years 2016-2026
Table 10-1: RNAi reagent, technology and service companies
Table 10-2: Pharmaceutical companies using RNAi for drug discovery and development
Table 10-3: Biotechnology companies using RNAi for drug discovery and development
Table 10-4: Companies developing RNAi-based therapeutic products
Table 10-5: Major players in RNAi
Table 10-6: Proprietary reagents of ImuThes
Table 10-7: Product pipeline of Silence Therapeutics
Table 10-8: Collaborations in RNAi technologies

List of Figures
Figure 1-1: Relationship of DNA, RNA and protein in the cell
Figure 1-2: Schematic of suppression of gene expression by RNAi
Figure 1-3: Relation of RNAi to epigenetic mechanisms
Figure 2-1: Overview of ShortCut RNAi Kit
Figure 2-2: Gene silencing by RNAi induced with ddRNAi
Figure 2-3: saRNA for targeted gene activation
Figure 3-1: A schematic miRNA pathway
Figure 3-2: Molecular mechanisms of miRNA generation
Figure 3-3: miRNA alternations in neurodegenerative diseases
Figure 4-1: RNA aptamer-mediated targeted RNAi delivery in cancer therapy
Figure 7-1: Targeting disease by RNAi
Figure 7-2: Mechanism of action of inclisiran
Figure 7-3: Role of RNAi in personalized medicine
Figure 8-1: Problems with use of synthetic siRNAs and measures to prevent them
Figure 9-1: Unmet needs in RNAi technologies

Note: Product cover images may vary from those shown
3 of 4
Professor K. K. Jain is a neurologist/neurosurgeon by training and has been working in the biotechnology/biopharmaceuticals industry for several years. He received graduate training in both Europe and USA, has held academic positions in several countries and is a Fellow of the Faculty of Pharmaceutical Medicine of the Royal Colleges of UK. Currently he is a consultant at Jain PharmaBiotech. Prof. Jain is the author of 415 publications including 16 books (2 as editor) and 48 special reports, which have covered important areas in biotechnology, gene therapy and biopharmaceuticals.
Note: Product cover images may vary from those shown
4 of 4
Note: Product cover images may vary from those shown