FlashRNA® as a proven solution for Gene Editing
FlashRNA® can be used as an All-in-One system for a transient expression of gene editing systems. It has been used in many types of primary cells, from stem cells to immune cells, as well as human induced pluripotent stem cells (iPSCs), showing an efficient gene editing without compromising cell viability or the differentiation capacity of stem cells.
A proven efficiency for various editing approaches, from CRISPR/Cas9 to base editing
FlashRNA® as an All-in-One system for a transient expression of the guide RNAs and nuclease at the same time : One single step for knocking out your target gene(s)
Thanks to its transient nature, FlashRNA® is perfectly suited for CRISPR/Cas9 delivery, allowing the expression of the gene editing machinery just long enough to generate an efficient editing, while avoiding off-target effects than could result from a too long Cas9 expression.
FlashRNA® packages all components of the CRISPR/Cas9 system
Short-term and transient expression of nuclease using FlashRNA® : almost undetectable after 3 days
Cas9 mRNA level in hiPSC was monitored by real-time qPCR for 7 days, showing the rapid mRNA decline after FlashRNA® transduction, at different doses.
FlashRNA® is a tool of choice for efficient and safe editing into sensitive cells
Thanks to a gentle cell penetration and a high particle concentration, FlashRNA® can efficiently be used for gene delivery into iPSC, ensuring cells viability and maintaining their original phenotype and differentiation capacity.
FlashRNA® editing efficiency depends on gene accessibility and inherent sgRNA potency.
Reference: Mianné J, Nasri A, Nguyen Van C, Bourguignon C, Fieldès M, Ahmed E, Duthoit C, Martin N, Parrinello H, Louis A, Iché A, Gayon R, Samain F, Lamouroux L, Bouillé P, Bourdin A, Assou S, De Vos J. Efficient CRISPR/Cas9-mediated gene knockout and interallelic gene conversion in human induced pluripotent stem cells using non-integrative bacteriophage-chimeric retrovirus-like particles. BMC Biol 20, 8. 2022. https://doi.org/10.1186/s12915-021-01214.
Thanks to its high payload capacity (up to 10 kb in length), FlashRNA® can be used for the delivery of other editing systems, like base editors
FlashRNA® used for transient expression of an adenine base editor corrects the Hutchinson-Gilford progeria syndrome mutation and improves the skin phenotype in mice.
Project conducted in the Karolinska Institute, Sweden.
Reference: Whisenant, D., Lim, K., Revêchon, G., Yao, H., Bergo, M. O., Machtel, P., … & Eriksson, M. Transient expression of an adenine base editor corrects the Hutchinson-Gilford progeria syndrome mutation and improves the skin phenotype in mice. Nat Commun 13, 3068. 2022. https://doi.org/10.1038/s41467-022-30800-y
FlashRNA® for exon skipping and for gene editing in vivo
A highly promising therapeutic strategy to repair the defective dystrophin (DMD) gene
Duchenne disease : No cure, and current pre-clinical strategies use AAVs (DNA delivery) to deliver gene editing systems. Transient expression of gene editing enzyme minimizes the off-target activity while allowing the restoration of DMD activity using FlashRNA®
Collaboration with Institut Clinique de la Souris (ICS), France
Accelerate your in vivo models thanks to in vivo delivery of a recombinase directly into FloxP animal models
Intra muscular delivery of FlashRNA® expressing the Cre recombinase into the mT/mG reporter mice allows an efficient recombinase expression into the mouse quadriceps, resulting in a high deletion efficiency (as shown by quadriceps histology with GFP immuno-staining (green) 2 weeks after FlashRNA® administration), with no excision in other organs (not shown)
Collaboration with PHENOMIN-Institut Clinique de la Souris (ICS)
FAQ
What types of RNA can FlashRNA® deliver?
FlashRNA® can deliver various RNA types, including mRNAs, guide RNAs, coding and non-coding RNAs. For example for gene editing, Cas9 mRNA and single guide RNAs can be packaged within the same particle.
What is the duration of RNA expression with FlashRNA®?
The RNA expression with FlashRNA® is transient, meaning it lasts for a short period. The duration of expression is related to the half-life of the protein being expressed. Typically, for a short half-life reporter, protein expression is detectable from 4 hours post-transduction and reaches a peak after around 24 hours, before decreasing. For a long half-life reporter, expression can be detected up to 7 days after transduction.
Is there generation of DNA after RNA transfer by FlashRNA®?
No, the delivered RNAs are directly used by the target cell. There is no possibility of reverse transcription nor integration, as the LTRs and integrase have been deleted.
What is the maximum RNA payload capacity of FlashRNA® ?
FlashRNA® technology supports a high payload capacity, enabling the delivery of both small and large RNAs. Specifically, it can deliver multiple RNA molecules, including large RNAs up to 10 kilobases (kb) in length. This makes FlashRNA® suitable for complex applications such as cell reprogramming, gene editing, and immunotherapy, where the simultaneous delivery of several and/or large RNA molecules is required.
Is FlashRNA® already used in therapy?
Yes, FlashRNA® has been produced as a Drug Product, to be used for a First-in-Human phase 1/2 clinical trial in 2025 (LymphARN trial, to treat secondary lymphedema).
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