ArticleMolecular therapy. Methods & clinical development2017
Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs.
Article in Molecular therapy. Methods & clinical development, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed.
- AAV-mediated FGF21 gene therapy promotes health span extension by whole-body tissue-specific adaptations.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Current Situation on Diabetes Management: New Weapons Fighting the Disease in 2025.Current drug targets · 2026Review
- The Role of Gene Therapy and RNA-Based Therapeutic Strategies in Diabetes.International journal of molecular sciences · 2025Review
- Maturity-Onset Diabetes of the Young 10 (MODY10): A Comprehensive Review of Genetics, Clinical Features, and Therapeutic Advances.International journal of molecular sciences · 2025Review
- Review
- Cell Therapies and Gene Therapy for Diabetes: Current Progress.Current diabetes reviews · 2025Review
- Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- The promising potential of gene therapy for diabetes mellitus.Nature reviews. Endocrinology · 2024Article
- Advances and challenges of the cell-based therapies among diabetic patients.Journal of translational medicine · 2024Review
- Current views on etiology, diagnosis, epidemiology and gene therapy of maturity onset diabetes in the young.Frontiers in endocrinology · 2024Review
- Research progress on the mechanism of TCM regulating intestinal microbiota in the treatment of DM mellitus.Frontiers in endocrinology · 2024Review
- Generation of a Beta-Cell Transplant Animal Model of Diabetes Using CRISPR Technology.Advances in experimental medicine and biology · 2023Article
- A Mini-Review of Current Treatment Approaches and Gene Therapy as Potential Interventions for Diabetes Mellitus Types 1.Advanced biomedical research · 2023Review
- Emerging Treatment Strategies for Diabetes Mellitus and Associated Complications: An Update.Pharmaceutics · 2021Review
- Diagnosis and treatment of type 1 diabetes at the dawn of the personalized medicine era.Journal of translational medicine · 2021Review
- From Mesenchymal Stromal/Stem Cells to Insulin-Producing Cells: Progress and Challenges.Stem cell reviews and reports · 2020Review
- A Cure for Sanfilippo Syndrome? A Summary of Current Therapeutic Approaches and their Promise.Medical research archives · 2020Article
- Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.Human gene therapy · 2019Article
- Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.Molecular therapy. Methods & clinical development · 2019Article
- FGF21 gene therapy as treatment for obesity and insulin resistance.EMBO molecular medicine · 2018Article
Corrections and comments
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Authors and funding
17 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Diabetes is a complex metabolic disease that exposes patients to the deleterious effects of hyperglycemia on various organs. Achievement of normoglycemia with exogenous insulin treatment requires the use of high doses of hormone, which increases the risk of life-threatening hypoglycemic episodes. We developed a gene therapy approach to control diabetic hyperglycemia based on co-expression of the insulin and glucokinase genes in skeletal muscle. Previous studies proved the feasibility of gene delivery to large diabetic animals with adeno-associated viral (AAV) vectors. Here, we report the long-term (∼8 years) follow-up after a single administration of therapeutic vectors to diabetic dogs. Successful, multi-year control of glycemia was achieved without the need of supplementation with exogenous insulin. Metabolic correction was demonstrated through normalization of serum levels of fructosamine, triglycerides, and cholesterol and remarkable improvement in the response to an oral glucose challenge. The persistence of vector genomes and therapeutic transgene expression years after vector delivery was documented in multiple samples from treated muscles, which showed normal morphology. Thus, this study demonstrates the long-term efficacy and safety of insulin and glucokinase gene transfer in large animals and especially the ability of the system to respond to the changes in metabolic needs as animals grow older.
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