Evidence map›Paper›PMID 41216426›Full record

ArticleMolecular therapy. Nucleic acids2025

A novel gene therapy platform for the treatment of type 2 diabetes and obesity.

Jared Lourie, Alex Goraltchouk, Judith M Hollander, Nicolas J Berger, H Grace Rosen, Atsutaro A Fujishiro, Francesco Luppino, Alexey Seregin, Luke Rhym, Kai Zou

Abstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
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.

Jared LourieDepartment of Exercise and Health Sciences, University of Massachusetts Boston, Boston, MA, USA.
Alex GoraltchoukRemedium Bio, Inc., Needham, MA, USA.
Judith M HollanderRemedium Bio, Inc., Needham, MA, USA.
Nicolas J BergerDepartment of Exercise and Health Sciences, University of Massachusetts Boston, Boston, MA, USA.
H Grace RosenDepartment of Biology, University of Massachusetts Boston, Boston, MA, USA.
Atsutaro A FujishiroDepartment of Exercise and Health Sciences, University of Massachusetts Boston, Boston, MA, USA.
Francesco LuppinoRemedium Bio, Inc., Needham, MA, USA.
Alexey SereginRemedium Bio, Inc., Needham, MA, USA.
Luke RhymRemedium Bio, Inc., Needham, MA, USA.
Kai ZouDepartment of Exercise and Health Sciences, University of Massachusetts Boston, Boston, MA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Diabetes and obesity are a growing healthcare and socioeconomic crisis of unprecedented proportions. Driven in part by the westernization of diets around the world, the prevalence of individuals with type 2 diabetes alone is projected to exceed 1.3 billion by 2050. Glucagon-like peptide 1 (GLP1) receptor agonists offer transformative therapeutic potential but are challenged by gastrointestinal side effects due to pharmacokinetic spikes from repeated injections, resulting in discontinuation rates which approach 70% by the first anniversary of treatment initiation. To overcome the repeat-injection nature of current GLP1 therapies, we developed a novel, subcutaneously injectable, lipid nanoparticle-based DNA delivery system to administer and express exendin 4 (EX4) or a modified natural GLP1 peptide in obese diabetic mice. The treatment was well tolerated, durable, and the transgene remained localized to the subcutaneous area of injection for over 6 months. The expressed EX4 and GLP1 promoted weight loss and decreased food intake, while reducing insulin resistance and improving glycemic control. Changes in the transcriptomic profile indicated that efficacy was mediated via the GLP1 receptor pathway, and blood-based biomarkers of liver and pancreatic function, systemic inflammation, and muscle injury confirmed that the treatment was systemically well tolerated.

Indexed as

DNAexendin 4gene therapyGLP1 RAlipid nanoparticlesMT: Delivery Strategiesobesitytype 2 diabetes

Identifiers

PMID41216426
PMCPMC12597059

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.