Gene Therapy on Type 1 Diabetes Mellitus: An Overview of Contemporary Treatment Strategies for Type 1 Diabetes Mellitus

The autoimmune disease known as type 1 diabetes mellitus (T1DM) is typified by the death of pancreatic β-cells, which results in insulin insufficiency and a permanent reliance on exogenous insulin. One possible method for restoring endogenous insulin production and maybe curing type 1 diabetes is gene therapy. This tactic uses stem cell-based gene delivery, viral and non-viral vectors, and gene editing methods like CRISPR-Cas9 to either preserve already-existing β-cells, replace lost β-cells, or add insulin-producing cells from different sources. Recent developments concentrate on immune regulation to guarantee long-term safety and efficacy while halting more β-cell death. Ongoing preclinical and clinical experiments are moving gene therapy closer to clinical implementation, despite obstacles like accurate gene targeting, immunological responses, and long-term integration. This review explores current progress, challenges, and future directions in gene therapy for T1DM, highlighting its potential as a transformative treatment.

Publication Details

Journal
Zenodo (CERN European Organization for Nuclear Research)
Published
2025-04-11
DOI
https://doi.org/10.5281/zenodo.15199136
Primary Topic
Pancreatic function and diabetes
Type
article
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article

Gene Therapy on Type 1 Diabetes Mellitus: An Overview of Contemporary Treatment Strategies for Type 1 Diabetes Mellitus

Zenodo (CERN European Organization for Nuclear Research)
Pancreatic function and diabetes
article

Gene Therapy on Type 1 Diabetes Mellitus: An Overview of Contemporary Treatment Strategies for Type 1 Diabetes Mellitus

article en

Abstract

The autoimmune disease known as type 1 diabetes mellitus (T1DM) is typified by the death of pancreatic β-cells, which results in insulin insufficiency and a permanent reliance on exogenous insulin. One possible method for restoring endogenous insulin production and maybe curing type 1 diabetes is gene therapy. This tactic uses stem cell-based gene delivery, viral and non-viral vectors, and gene editing methods like CRISPR-Cas9 to either preserve already-existing β-cells, replace lost β-cells, or add insulin-producing cells from different sources. Recent developments concentrate on immune regulation to guarantee long-term safety and efficacy while halting more β-cell death. Ongoing preclinical and clinical experiments are moving gene therapy closer to clinical implementation, despite obstacles like accurate gene targeting, immunological responses, and long-term integration. This review explores current progress, challenges, and future directions in gene therapy for T1DM, highlighting its potential as a transformative treatment.

Zenodo (CERN European Organization for Nuclear Research)
Good health and well-being
Openalex Percentile: Top 35%
Pancreatic function and diabetes
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Gene Therapy on Type 1 Diabetes Mellitus: An Overview of Contemporary Treatment Strategies for Type 1 Diabetes Mellitus · Zenodo (CERN European Organization for Nuclear Research) (2025) | TGRS Research Map | TGRS