Evidence map›Paper›PMID 41888818›Full record

ArticleBMC biotechnology2026

Identification of computationally designed skeletal and cardiac promoters with high specificity across AAV delivery routes.

Nicole Zielinska, Erin L Howard, Brenna A Y Stevens, Melanie M Goens, Cici Yang, Elena S B Campbell, Madison E Hughes, Yanlong Pei, Dinghai Zheng, D Benjamin Gordon and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in BMC biotechnology, 2026. 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

15 authors.

Nicole ZielinskaDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Erin L HowardDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Brenna A Y StevensDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Melanie M GoensDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Cici YangDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Elena S B CampbellDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Madison E HughesDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Yanlong PeiDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Dinghai ZhengAsimov, Boston, MA, USA.
D Benjamin GordonAsimov, Boston, MA, USA.
Alec A K NielsenAsimov, Boston, MA, USA.
Raja R SrinivasAsimov, Boston, MA, USA.
Jeff L CaswellDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada.
Luis G ArroyoDepartment of Clinical Studies, Ontario Veterinary College, Guelph, Ontario, Canada.
Sarah K WoottonDepartment of Pathobiology, University of Guelph, Guelph, Ontario, Canada. kwootton@uoguelph.ca.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPrecise control of transgene expression is essential for safe and effective AAV gene therapies. While capsid engineering has advanced tissue targeting, progress in developing regulatory elements that confine expression to specific tissues has lagged. This gap limits the ability to minimize off-target expression and associated safety risks, emphasizing the need for improved tissue-specific regulatory control.

resultsHere, we evaluated 12 computationally designed tissue-specific promoters generated by Asimov’s proprietary algorithm. Promoters driving a heat-stable human placental alkaline phosphatase reporter gene were packaged into AAV6·2FF and administered to C57BL/6 mice intranasally, intramuscularly, and intraperitoneally. Expression was assessed macroscopically, microscopically, enzymatically and by vector genome quantification. We identified two strictly skeletal muscle-specific promoters independent of the AAV capsid or route of administration used, and two cardiac-specific promoters when administered intraperitoneally.

conclusionsThese findings demonstrate that computational promoter design can yield elements with great strength and precision that enable safer and more targeted AAV gene therapies and offer a broadly applicable strategy for tissue-specific expression in treating various monogenic diseases.

Indexed as

DependovirusGenetic VectorsMuscle, SkeletalMyocardiumPromoter Regions, GeneticAlgorithmsAlkaline PhosphataseAnimalsGenes, ReporterGenetic TherapyGene Transfer TechniquesGPI-Linked ProteinsHumansIsoenzymesMiceMice, Inbred C57BLAlkaline Phosphatasealkaline phosphatase, placentalGPI-Linked ProteinsIsoenzymesAAVAlgorithmCardiac musclePromotersSkeletal muscleTargetedTissue-specific

Identifiers

PMID41888818
PMCPMC13147773

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.