Rational design of a compact CRISPR-Cas9 activator for AAV-mediated delivery
Open accessAbstract
Akin to Zinc Finger and Transcription Activator Like Effector based transcriptional modulators, nuclease-null CRISPR-Cas9 provides a groundbreaking programmable DNA binding platform, begetting an arsenal of targetable regulators for transcriptional and epigenetic perturbation, by either directly tethering, or recruiting, transcription enhancing effectors to either component of the Cas9/guide RNA complex. Application of these programmable regulators is now gaining traction for the modulation of disease-causing genes or activation of therapeutic genes, in vivo . Adeno-Associated Virus (AAV) is an optimal delivery vehicle for in vivo delivery of such regulators to adult somatic tissue, due to the efficacy of viral delivery with minimal concerns about immunogenicity or integration. However, present Cas9 activator systems are notably beyond the packaging capacity of a single AAV delivery vector capsid. Here, we engineer a compact CRISPR-Cas9 activator for convenient AAV-mediated delivery. We validate efficacy of the CRISPR-Cas9 transcriptional activation using AAV delivery in several cell lines.
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Two sNRP-1 polyA sequences were less suitable for specific transcripts compared to bGHpolyA or spA.
“Yet, the two sNRP-1 polyA were less suitable for specific transcripts compared to bGHpolyA or spA [167].”
From Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays (2020) · cites this paper as [167]
Figure 7 (section ‘AAV transduction in cell lines’; page unavailable) reports fewer dSaCas9 activator transcripts with both 1X and 2X sNRP-1 than with bGHR polyA. It does not establish that they were less suitable than synthetic polyA (spA), and target-gene expression was unchanged or higher despite the lower activator-transcript counts. (LLM, conf=0.86)
Experimental — accuracy not yet measuredA modified rAAV-vector version with a full-length CMV promoter and the bGHpolyA efficiently upregulated a gene (Actc1, Neurog2, or Hbb) upon infection of N2A neuron derived cells by 50-150× in vitro.
“A modified rAAV-vector version with a full-length CMV promoter and the bGHpolyA efficiently upregulated a gene (Actc1, Neurog2, or Hbb) upon infection of N2A neuron derived cells by 50-150× in vitro [167].”
From Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays (2020) · cites this paper as [167]
The ‘Activation of endogenous genes with single vector system’ section reports activation of Actc1, Neurog2, and Hbb in N2A cells, but those experiments used transfection; it assigns the full-length CMV promoter and bGHR signal to the comparator vector, not the new compact activator. The ‘Delivery of single vector activator via AAV to activate target genes in vitro’ section reports 54–149-fold Hbb upregulation after AAV delivery in three other cell lines. It says the virus was toxic to Neuro-2A cells, preventing collection of expression data there. (LLM, conf=0.90)
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- Authors
- Vora, S., Cheng, J., Xiao, R., VanDusen, N. J., Quintino, L., Pu, W. T., Vandenberghe, L. H., Chavez, A., & Church, G.
- Affiliations
- Wyss Institute for Biologically Inspired Engineering (US)
- Massachusetts Eye and Ear Infirmary (US)
- Boston Children's Hospital (US)
- Broad Institute (US)
- ORCID
- Venue
- bioRxiv (Cold Spring Harbor Laboratory)
- Publisher
- openRxiv
- Published
- 2018-04-15
- OpenAlex
- W2797286246
- Cited by
- 42
- References
- 38
- Citations by year
- 2026: 2 · 2025: 11 · 2024: 10 · 2023: 4 · 2022: 5 · 2021: 4 · 2020: 4 · 2019: 1 · 2018: 1
- Type
- posted content
- Language
- en
- Open access
- green
- doi.org
- Locations
- bioRxiv (Cold Spring Harbor Laboratory) (acceptedVersion)
- Lund University Publications (Lund University) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- CRISPR/Cas9, transcriptional activation, adeno-associated virus vectors, adeno-associated virus, gene delivery, guide RNA, somatic gene therapy, in vivo gene regulation
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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