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

doi:10.1016/j.omtn.2025.102739

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

Exercise & Health Science

University of Massachusetts Boston
Inc.

Research output: Contribution to journal › Article › peer-review

Abstract

While GLP1 injections have revolutionized treatment of diabetes and obesity, current approaches suffer from poor tolerability and high discontinuation. Goraltchouk and colleagues developed a gene therapy treatment delivering GLP1 DNA to subcutaneous fat cells for long-term therapeutic expression. Treatment was well tolerated, improving weight, glycemia, and insulin sensitivity in mice.

Original language	English
Article number	102739
Journal	Molecular Therapy Nucleic Acids
Volume	36
Issue number	4
DOIs	https://doi.org/10.1016/j.omtn.2025.102739
State	Published - Dec 9 2025

ASJC Scopus Subject Areas

Molecular Medicine
Drug Discovery

Keywords

DNA
exendin 4
gene therapy
GLP1 RA
lipid nanoparticles
MT: Delivery Strategies
obesity
type 2 diabetes

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10.1016/j.omtn.2025.102739

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abstract = "While GLP1 injections have revolutionized treatment of diabetes and obesity, current approaches suffer from poor tolerability and high discontinuation. Goraltchouk and colleagues developed a gene therapy treatment delivering GLP1 DNA to subcutaneous fat cells for long-term therapeutic expression. Treatment was well tolerated, improving weight, glycemia, and insulin sensitivity in mice.",

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AU - Lourie, Jared

AU - Goraltchouk, Alex

AU - Hollander, Judith M.

AU - Berger, Nicolas J.

AU - Rosen, H. Grace

AU - Fujishiro, Atsutaro A.

AU - Luppino, Francesco

AU - Seregin, Alexey

AU - Rhym, Luke

AU - Zou, Kai

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AB - While GLP1 injections have revolutionized treatment of diabetes and obesity, current approaches suffer from poor tolerability and high discontinuation. Goraltchouk and colleagues developed a gene therapy treatment delivering GLP1 DNA to subcutaneous fat cells for long-term therapeutic expression. Treatment was well tolerated, improving weight, glycemia, and insulin sensitivity in mice.

KW - DNA

KW - exendin 4

KW - gene therapy

KW - GLP1 RA

KW - lipid nanoparticles

KW - MT: Delivery Strategies

KW - obesity

KW - type 2 diabetes

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

Abstract

ASJC Scopus Subject Areas

Keywords

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