Evidence map›Paper›PMID 38981468›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2024

Sleeping Beauty mRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency.

Philip M Zakas, Sharon C Cunningham, Ann Doherty, Eva B van Dijk, Raed Ibraheim, Stephanie Yu, Befikadu D Mekonnen, Brendan Lang, Elizabeth J English, Gang Sun and 24 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing 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

14 citing papers in PubMed.

  1. Review
  2. Review
  3. RNA-Based Therapies for Inherited Metabolic Disorders.Journal of inherited metabolic disease · 2026
    Review
  4. Review
  5. Review
  6. Review
  7. Article
  8. Review
  9. mAdvanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Article
  10. The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  11. Article
  12. Review
  13. Article
  14. Hybrid non-viral and viral delivery strategy achieves potent gene editing in growing livers with reduced viral dosage.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

34 authors.

Philip M ZakasTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Sharon C CunninghamGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.
Ann DohertyTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Eva B van DijkGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.
Raed IbraheimTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Stephanie YuTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Befikadu D MekonnenTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Brendan LangTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Elizabeth J EnglishTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Gang SunTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Miles C DuncanTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Matthew S BenczkowskiTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Robert C AltshulerTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Malvenderjit Jagjit SinghTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Emily S KibblerTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Gulen Y TongaTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Zi Jun WangTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Z Jane WangTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Guangde LiTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Ding AnTessera Therapeutics, Inc., Somerville, MA 02143, USA.
James B RottmanTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Yashvi BhavsarTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Cormac PurcellTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Rachit JainTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Ryan AlberryTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Nathaniel RoquetTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Yanfang FuTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Robert J CitorikTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Jacob R RubensTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Michael C HolmesTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Cecilia Cotta-RamusinoTessera Therapeutics, Inc., Somerville, MA 02143, USA.
William QuerbesTessera Therapeutics, Inc., Somerville, MA 02143, USA.
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia; Discipline of Child and Adolescent Health, University of Sydney, Westmead, NSW 2145, Australia. Electronic address: ian.alexander@health.nsw.gov.au.
William E SalomonTessera Therapeutics, Inc., Somerville, MA 02143, USA. Electronic address: wsalomon@tesseratx.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recombinant adeno-associated virus (rAAV) vector gene delivery systems have demonstrated great promise in clinical trials but continue to face durability and dose-related challenges. Unlike rAAV gene therapy, integrating gene addition approaches can provide curative expression in mitotically active cells and pediatric populations. We explored a novel in vivo delivery approach based on an engineered transposase, Sleeping Beauty (SB100X), delivered as an mRNA within a lipid nanoparticle (LNP), in combination with an rAAV-delivered transposable transgene. This combinatorial approach achieved correction of ornithine transcarbamylase deficiency in the neonatal Spf

Indexed as

DependovirusGenetic VectorsHepatocytesNanoparticlesRNA, MessengerTransgenesTransposasesAnimalsDisease Models, AnimalGene ExpressionGenetic TherapyGene Transfer TechniquesHumansLipidsLiposomesMiceLipid NanoparticlesLipidsLiposomesOrnithine CarbamoyltransferaseRNA, Messengersleeping beauty transposase, humanTransposasesadeno-associated virus vectorgene additionlipid nanoparticlemacaquemRNA liver deliveryornithine transcarbamylasepartial hepatectomySleeping Beauty transposasetransposition

Identifiers

PMID38981468
PMCPMC11489535

What OpenQuestion holds

Textmetadata
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.