Evidence map›Paper›PMID 39416069›Full record

ArticlebioRxiv : the preprint server for biology2025

Tunable, proteolytic dosage control of CRISPR-Cas systems enables precise gene therapy for dosage sensitive disorders.

Noa Katz, Connie An, Yu-Ju Lee, Josh Tycko, Meng Zhang, Jeewoo Kang, Lacramioara Bintu, Michael C Bassik, Wei-Hsiang Huang, Xiaojing J Gao

Abstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in bioRxiv : the preprint server for biology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Noa KatzDepartment of Chemical Engineering, Stanford University, Stanford, CA, USA.
Connie AnDepartment of Chemical Engineering, Stanford University, Stanford, CA, USA.ORCID 0000-0003-3977-1497
Yu-Ju LeeDepartment of Neurology & Neurosurgery, Centre for Research in Neuroscience, McGill University, Montréal, QC H3G 1A3, Canada.
Josh TyckoDepartment of Genetics, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0002-4108-0575
Meng ZhangDepartment of Chemical Engineering, Stanford University, Stanford, CA, USA.
Jeewoo KangNeurosciences Interdepartmental Program, Stanford University, Stanford, CA, 94305, USA.
Lacramioara BintuDepartment of Bioengineering, Stanford University, Stanford, CA, USA.ORCID 0000-0001-5443-6633
Michael C BassikDepartment of Genetics, Stanford University, Stanford, CA 94305, USA.
Wei-Hsiang HuangDepartment of Neurology & Neurosurgery, Centre for Research in Neuroscience, McGill University, Montréal, QC H3G 1A3, Canada.
Xiaojing J GaoDepartment of Chemical Engineering, Stanford University, Stanford, CA, USA.

Funding

Synthetic DNA-free Circuits for “Scarless” Programming of Mammalian CellsR00EB027723 · NIBIB · STANFORD UNIVERSITY · PI GAO, XIAOJING J · 2020 to 2022
$747k
NIBIB NIH HHS R00 EB027723
6 · The paper itself

Abstract

The ability to modulate gene expression through modular and universal genetic tools like CRISPR-Cas has greatly advanced gene therapy for therapeutics and basic science. Yet, the inherent stochasticity of delivery methods cause variation in target gene expression at the single-cell level, limiting their applicability in systems that require more precise expression. Thus, we implement a modular incoherent feedforward loop based on proteolytic cleavage of Cas to reduce gene expression variability against the variability of vector delivery. We target a genome-integrated marker and demonstrate dosage control of gene activation and repression, post-delivery tuning, and RNA-based compatibility of the system. To illustrate therapeutic relevance, we target the gene

Identifiers

PMID39416069
PMCPMC11482798

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.