RNAiONE™ algorithmic identification of shRNA sequences

SIRION Biotech offers RNAiONE™ as a guaranteed path to define the most effective small hairpin RNA (shRNA) sequences for target gene identification and validation which can be further translated into SIRION’s viral vector platform.

RNAiONE™ provides one solution for two challenges by identifying the most optimal shRNA sequence, and engineering a viral vector with the best performing validated shRNA. RNAiONE™ serves for effective gene knockdown with success rates above 90%.

Over the years SIRION Biotech has developed its base technology to the point where in 80% of recent projects it managed to identify targeted shRNA sequences with knockdown results of above 90%.

Identifying elusive shRNA sequences for effective gene knockdown

A short hairpin RNA (shRNA) is an artificial RNA molecule that is used to silence target gene expression via RNA interference (RNAi). shRNA has the following mechanism of action: shRNA binds to the complementary target mRNA in cell, inactivates or degrades the target mRNA leading to gene silencing. In gene therapy this can be relevant to certain types of cancer, where specific mRNAs were shown to be involved.

Expression of shRNA in cells is reached by delivery of shRNA to cells using viral vectors. Different viral vectors can be used to bring shRNA in cells including adeno-associated viruses (AAV), adenoviruses, and lentiviruses. AAV and adenoviruses do not integrate into genome, so gene expression remains relatively transient. Lentivirus provide a possibility to obtain stable gene expression due to viral genome integration allowing long-term knockdown of the target gene.

RNAiONE™ solution for uncovering best possible shRNA sequences

Designing and validation of the best performing shRNAs is essential for effective RNAi knockdown. The key challenge in designing of the effective shRNAs is to isolate the most optimal sequence from millions of possible combinations.

SIRION Biotech’s algorithm-based design of shRNA sequences allows successful identification of 10-15 most active shRNA sequences. The results are fully translatable to SIRION Biotech`s viral vector platforms and allow knockdown efficiency of more than 80 % on mRNA level.

RNAiONE™ can be applied whenever knockdowns are needed, including:

• Gene loss of function studies
• Gene expression silencing
• Target identification & validation for early point drug discovery (e.g. in oncology)
• Cell model generation

RNAiONE™ benefits: guaranteed results

The RNAiONE™ solution delivers a series of quantifiable benefits, including:

• • Knockdown guarantee of > 80% on mRNA level
  • Results are fully translatable into viral vector platforms
  • Full portfolio of RNAiONE compatible viral vector platforms (Lentivirus, Adenovirus, AAV)
  • RNAiONE validation of hard-to-knockdown targets with knock-down efficiency more than 90 %
  • Validated shRNA in less than three weeks
  • Documented project statistics
  • Customer Satisfaction Guarantee