ArticleMolecular therapy : the journal of the American Society of Gene Therapy2011
Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 papers.
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Who cites it
52 citing papers in PubMed, 92 citations in OpenAlex.
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- Treatment with Minicircle DNA Expressing a FGF23 Fragment in a Clinically relevant Mouse Model of X-Linked Hypophosphatemic Rickets.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Anticodon Edited Transfer RNAs (ACE-tRNAs) Encoded as Therapeutic Nonviral Minimal DNA Vectors.bioRxiv : the preprint server for biology · 2025Article
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- MicroRNA-206 as a potential cholesterol-lowering drug is superior to statins in mice.Journal of lipid research · 2024Article
- DNA Vaccines: Their Formulations, Engineering and Delivery.Vaccines · 2024Review
- Therapeutic effect of multiple functional minicircle vector encoding anti-CD25/IL-10/CXCR3 in allograft rejection model.The Korean journal of internal medicine · 2022Article
- Nucleic Acid Delivery from Granular Hydrogels.Advanced healthcare materials · 2022Article
- Advances in the Development and the Applications of Nonviral, Episomal Vectors for Gene Therapy.Human gene therapy · 2021Article
- A non-viral genome editing platform for site-specific insertion of large transgenes.Stem cell research & therapy · 2020Article
- Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Construction and Validation of a Novel Glycometabolism-Related Gene Signature Predicting Survival in Patients With Ovarian Cancer.Frontiers in genetics · 2020Article
- Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases.Frontiers in molecular biosciences · 2020Review
- Gene Therapy Today and Tomorrow.Diseases (Basel, Switzerland) · 2019Article
- Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.Genetics and molecular biology · 2019Article
- DNA Vaccines-How Far From Clinical Use?International journal of molecular sciences · 2018Review
- Nonviral Gene Therapy for Cancer: A Review.Diseases (Basel, Switzerland) · 2018Review
- Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Biomaterial-Guided Gene Delivery for Musculoskeletal Tissue Repair.Tissue engineering. Part B, Reviews · 2017Review
Corrections and comments
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Authors and funding
10 authors at 6 institutions in 2 countries.
Funding
Abstract
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease characterized by mutations to the α-L-iduronidase (IDUA) gene resulting in inactivation of the IDUA enzyme. The loss of IDUA protein results in the progressive accumulation of glycosaminoglycans within the lysosomes resulting in severe, multi-organ system pathology. Gene replacement strategies have relied on the use of viral or nonviral gene delivery systems. Drawbacks to these include laborious production procedures, poor efficacy due to plasmid-borne gene silencing, and the risk of insertional mutagenesis. This report demonstrates the efficacy of a nonintegrating, minicircle (MC) DNA vector that is resistant to epigenetic gene silencing in vivo. To achieve sustained expression of the immunogenic IDUA protein we investigated the use of a tissue-specific promoter in conjunction with microRNA target sequences. The inclusion of microRNA target sequences resulted in a slight improvement in long-term expression compared to their absence. However, immune modulation by costimulatory blockade was required and permitted for IDUA expression in MPS I mice that resulted in the biochemical correction of pathology in all of the organs analyzed. MC gene delivery combined with costimulatory pathway blockade maximizes safety, efficacy, and sustained gene expression and is a new approach in the treatment of lysosomal storage disease.
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