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 in
clinical development yet. The major use of RNAi reagents is in research but it
partially overlaps that of drug discovery and therapeutic development. It is
estimated to be $400 million currently in 2005,$850 million by the year 2010
and $1.2 billion by the year 2015. The value of the drug discovery market based
on RNAi can be assessed at $650 million in the year 2005, increasing to $1
billion in the year 2010 and $1.5 billion in 2015. Even if a few products get
into the market by the year 2010, this market will expand to $3.5 billion based
on revenues from sales of RNAi-based drugs and increase further to $5.9 billion
in 2015. Markets are also analyzed according to breakdown of technologies and
use of siRNAs, miRNAs, etc.
Profiles of 134 companies involved in developing RNAi technologies are
presented along with 120 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. Approximately 18 of these are developing
RNAi-based therapeutics. The bibliography contains selected 300 publications
that are cited in the report. The text is supplemented with 26 tables and 7
figures.
Topics Covered
0Executive Summary
1.Technologies for suppressing gene function
2.RNAi Technologies
3.MicroRNA
4.Methods of delivery in RNAi
6.RNAi in drug discovery
7.Therapeutic applications of RNAi
8.Safety, regulatory and patent issues
9.Markets for RNAi Technologies
10.Companies involved in RNAi Technologies
11 References
List of Tables
List of Figures
For more information visit http://www.researchandmarkets.com/reports/c7805
Senior Manager
Research and Markets
press@researchandmarkets.com
Fax: +353 1 4100 980
Issuers of news releases, not Targetwire, are solely responsible for the accuracy of the content.