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DESIGN

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Our workflow

Project workflow diagram showing weekly milestones

CRISPR/Cas13a Design

Selection of suitable Cas13a protein

The CRISPR/Cas13a system is the first step of our diagnostic approach, as it can be designed for the specific recognition of our microRNA biomarkers. It consists of two parts: Cas13a nuclease and CRISPR RNA (crRNA). The crRNA directs Cas13a to single-stranded RNA targets with high specificity through sequence complementarity, making the CRISPR/Cas13a system suitable for the recognition of our microRNA biomarkers. When Cas13a binds its RNA target, it becomes activated. Activated Cas13a not only cleaves the target RNA but also performs “collateral” cleavage of nearby non-target RNAs. This property allows the generation of detectable signals even at low biomarker concentrations. It also enables real-time monitoring and increases the sensitivity of detection. (1) To maximize sensitivity of our approach, we selected a Cas13a ortholog with high collateral cleavage activity. Through literature research, we found that among fifteen Cas13a orthologs, the ortholog from Leptotrichia wadei (LwCas13a) was the most active with high trans-cleavage activity. (2)

Design of suitable guideRNAs for both of our miRNAs targets

To design crRNAs for our CRISPR/Cas13a system, we based our approach on both our selected microRNA biomarker sequences and existing literature. Each crRNA consists of two key regions:

  • Direct Repeat (DR) (yellow) → The DR is a 36-nt sequence that folds into a stem–loop structure, which is essential for interaction with the Cas13a protein scaffold. For LwCas13a, the DR sequence is:

    GAUUUAGACUACCCCAAAAACGAAGGGGACUAAAAC (3)

    Illustration of the final gRNA1 structure
    Illustration of the final gRNA2 structure
  • Spacer (blue) → The spacer sequence is complementary to the target RNA. The optimal spacer length for LwCas13a is 28 nucleotides. However, since our target microRNAs are only 22 nucleotides long, we explored whether shorter spacers could still be effective. Literature indicates that LwCas13a retains cleavage activity with spacers as short as 20 nucleotides. We therefore designed 20–22 nt spacers complementary to our microRNAs, evaluated them in silico for predicted binding and structural compatibility, and selected the most promising candidates for experimental validation. READ MORE (2).

Plasmid construction for CRISPR/Cas13a expression

We constructed the plasmid for the expression of our CRISPR/Cas13a system by using as vector Cas_variants_hLwCas13a from the iGEM Distribution Kit 2025. This vector contained the gene of a variant of Cas13a, a high copy origin of replication and a gene for antibiotic resistance to Chloramphenicol. We designed the two fragments, that contained the gRNA genes each:

Fragment for gRNA1

Diagram showing plasmid fragment for gRNA1

Fragment for gRNA2

Diagram showing plasmid fragment for gRNA2

Our fragments contained:

BBa_J23101 and BBa_J23100: strong, constitutive promoters for the continuous expression of gRNAs and hLwCas13a protein respectively.

Bacterial terminator for the end of gRNA genes transcription

gRNA genes

Ribosome binding site downstream BBa_J23100 promoter for better Cas13a expression

6xHis Tag for subsequent protein purification

Transformation

After cloning, we inserted our plasmid into Rosetta 2(DE3)pLysS cells for the expression of our CRISPR/Cas13a system. This cell line is derived from E.coli BL21 and is suitable for the expression of proteins that contain codons rare for E.coli, thanks to the extra tRNAs encoded on a chloramphenicol-resistant plasmid (pRARE2). Since our expression vector also carries chloramphenicol resistance, the antibiotic selection pressure ensures maintenance of both the expression plasmid and the rare-tRNA helper plasmid. 24 hours after transformation we isolated DNA from single colonies to verify whether our plasmid was right.

Protein expression

We expressed our system by inoculating bacterial colonies with our plasmid into liquid LB. We performed one small starter culture, expecting overnight growth. The day after, we added the culture into bigger LB quantity to ensure higher protein yield. The use of constitutive promoters allowed for continuous expression of LwaCas13a without the need for induction.

Protein purification

By adding a 6xHis affinity tag to the N-terminal end of our Cas13a protein we aimed to ensure purification by using HisPur Ni-NTA columns. A 3 amino acid linker between the His Tag and the protein was added for subsequent cleavage of the His Tag after purification using a TEV protease. The Ribosome Binding site ensures that translation of Cas13a will occur in the right place. We verified the results of the purification with a 10% SDS PAGE electrophoresis.

Catalytic Hairpin Assembly Design

Hairpin probes design

Hairpin probes design

In the Catalytic Hairpin Assembly participate two partially complementary DNA hairpins, DNA hairpin probe 1 (H1) & DNA hairpin probe 2 (H2) and one single-stranded oligonucleotide (Initiator). The hairpin motif of H1 contains three concatenated domains and each domain has a special nucleation site, called toehold.

  • a domain with toehold at
  • b domain with toehold bt
  • c domain with toehold ct (3)
H1 Diagram of hairpin probe H1
H2 Diagram of hairpin probe H2

The hairpin motif of H2 contains two continuous domains, each of them has also a toehold. These domains are designed to be complementary to two of the H1 domains: *=complementary

a* domain with toehold at* (complementary to H1 domain a)

b* domain with toehold bt* (complementary to H1 domain b)

The hairpin probes in our system were designed based on the initiator sequence. The initiator is a single-stranded oligonucleotide that serves to trigger the Catalytic Hairpin Assembly (CHA) reaction. More specifically, the initiator is part of a non-specific DNA hairpin 0 for CRISPR/Cas13a and is released after its activation upon biomarker recognition and the subsequent collateral cleavage activity. Hairpin H0 differs from H1 and H2. It has a DNA stem and an RNA loop that serves as a collateral cleavage site for Cas13a. To maximize Cas13a activity, the loop contains a poly-U region, as LwCas13a collateral cleavage activity is enhanced on UU base motifs (5).

Cartoon showing domains of hairpin H0 Sequence diagram of hairpin H0

The initiator is released after cleavage of H0 in the polyuracil region inside its loop. The initiator binds to the complementary a domain of H1 via its at* toehold. This nucleation triggers a branch migration that opens H1.The opened H1 has now the domains b and c exposed,along with the toeholds bt and ct. The bt toehold can then serve as binding site for hairpin H2.A disassembly reaction occurs when single-stranded domain a* of H2 initiates a branch migration that displaces the initiator from H1. (3)

Initiator sequence diagram Annotated sequence diagram of hairpin H1 Annotated sequence diagram of hairpin H2

H2 design

The sequence of H2 probe was designed based on H1, with two complementary domains (a* and b* domain of H2 complementary to a and b domain of H1). Domain a* overlaps with the initiator-binding site on H1, ensuring that once H2 binds, the initiator is displaced and recycled. The H1–H2 duplex is designed to be more stable than the ternary Initiator–H1–H2 complex, ensuring efficient turnover. (6)

Sequence adjustments

GC content and nucleotide lengths were tuned to balance thermodynamic stability and complementarity, rather than strictly following conventional design rules. (6)

H1 hairpin probe

Toehold: 9nt, <40% GC content

Stem: 11nt, close to the optimal of 12nt. It has a high GC content > 60%

Loop: 10nt, <40% GC content

Modification: 3’ sulfide group for Au electrode immobilization

H2 hairpin probe

Toehold: 7nt, < 40% GC content

Stem: 10nt

Loop: 11nt

Modification: 5’ methylene blue (MB) label

Each hairpin probe was analysed with NUPACK software to check the stability and thermodynamic properties. READ MORE

CRISPR/Cas13a reaction and Catalytic Hairpin Assembly

The CRISPR/Cas13a reaction was performed in a reaction buffer at 37 °C for 1 hour. The system included the Cas13a protein, H0 hairpin, and the target miRNA biomarker. Two separate reactions were conducted: one targeting miR-150-5p and the other miR-486-5p. Following the reaction, the resulting mixture was transferred into a solution containing H1 and H2 hairpin probes, allowing the Catalytic Hairpin Assembly (CHA) to proceed and form H1–H2 duplexes.

CRISPR/Cas13a and Catalytic Hairpin Assembly validation with electrophoresis

To assess the efficiency of both the CRISPR/Cas13a reaction and the Catalytic Hairpin Assembly (CHA), we performed 3% agarose and native PAGE electrophoresis. These analyses allowed us to verify whether the H1–H2 duplexes were formed following initiator release. In the presence of the target miRNAs, Cas13a activation cleaves the H0 hairpin, releasing the initiator that triggers CHA and drives H1–H2 duplex formation. The H1–H2 duplex appears as a distinct band at approximately 80 nucleotides, while unreacted hairpins (H1, H2, and H0) migrate at around 40 nucleotides. In the absence of the target miRNAs, no initiator is released, and no duplex band should be observed confirming the specificity of the CRISPR/Cas13a-CHA system.

Band Analysis

We analyzed the electrophoresis gels using Fiji software to quantify the band intensities corresponding to the H1–H2 duplexes. A strong duplex band indicates efficient CHA amplification and successful CRISPR/Cas13a activation. By comparing the band intensities in reactions with and without target miRNAs, we assessed both the specificity and efficiency of our system. A significant increase in the 80-nt duplex band intensity in the presence of target miRNAs confirms that the Cas13a-mediated initiator release effectively triggered the Catalytic Hairpin Assembly. Additionally, analyzing the relative duplex band intensity provides insight into the quantitative response of the system toward different miRNA concentrations.

References

  1. Jonathan S. Gootenberg et al. Nucleic acid detection with CRISPR-Cas13a/C2c2. Science 356, 438–442 (2017). https://doi.org/10.1126/science.aam9321
  2. Abudayyeh O.O., Gootenberg J.S., Essletzbichler P., Han S., Joung J., Belanto J.J., Verdine V., Cox D.B.T., Kellner M.J., Regev A., Lander E.S., Voytas D.F., Ting A.Y., Zhang F. RNA targeting with CRISPR-Cas13. Nature. 2017 Oct 12;550(7675):280-284. https://doi.org/10.1038/nature24049 PMID: 28976959; PMCID: PMC5706658.
  3. Kellner M.J., Koob J.G., Gootenberg J.S., Abudayyeh O.O., Zhang F. SHERLOCK: nucleic acid detection with CRISPR nucleases. Nat Protoc. 2019 Oct;14(10):2986-3012. https://doi.org/10.1038/s41596-019-0210-2 Erratum in: Nat Protoc. 2020 Mar;15(3):1311. https://doi.org/10.1038/s41596-020-0302-z
  4. Kulkarni A., Yu W., Moon A.S., Pandey A., Hanley K.A., Xu J. Programmable CRISPR interference for gene silencing using Cas13a in mosquitoes. J Genomics. 2020 Mar 1;8:30-36. https://doi.org/10.7150/jgen.43928 PMID: 32190127; PMCID: PMC7064736.
  5. Yin P., Choi H., Calvert C. et al. Programming biomolecular self-assembly pathways. Nature 451, 318–322 (2008). https://doi.org/10.1038/nature06451
  6. Zou M., Zhou M., Ma S., Zhang C., Xiao F., Wu H., GuliNaizhaer A., Yao Y., Chen Y., Cai S., Fan X., Zhao F., Wu G. Addition of dNTPs can improve the detection sensitivity of catalytic hairpin assembly. iScience. 2023 Mar 13;26(4):106390. https://doi.org/10.1016/j.isci.2023.106390 PMID: 37034995; PMCID: PMC10074145.
  7. Gootenberg J.S., Abudayyeh O.O., Kellner M.J., Joung J., Collins J.J., Zhang F. Multiplexed and portable nucleic acid detection platform with Cas13, Cas12a, and Csm6. Science. 2018 Apr 27;360(6387):439-444. https://doi.org/10.1126/science.aaq0179 PMID: 29449508; PMCID: PMC5961727.
  8. Wu J., Xu H., Hu F., Jiang Y., Fan B., Khan A., Sun Y., Di K., Gu X., Shen H., Li Z. CRISPR-Cas and catalytic hairpin assembly technology for target-initiated amplification detection of pancreatic cancer specific tsRNAs. Front Bioeng Biotechnol. 2023 May 3;11:1169424. https://doi.org/10.3389/fbioe.2023.1169424 PMID: 37207121; PMCID: PMC10188930.