ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Complete genetic correction of ips cells from Duchenne muscular dystrophy.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 119 papers.
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119 citing papers in PubMed, 254 citations in OpenAlex.
- Reconstitution of DNA fragments on HAC/MAC via the fragment-assembly system.Scientific reports · 2026Article
- Synthetic rewriting technologies in mammalian cells.Nature communications · 2026Review
- Application and Technical Challenges in Design, Cloning, and Transfer of Large DNA.Bioengineering (Basel, Switzerland) · 2023Review
- Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene.Molecular therapy. Nucleic acids · 2023Article
- Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up.Stem cell reviews and reports · 2023Article
- Human artificial chromosome carrying 3p21.3-p22.2 region suppresses hTERT transcription in oral cancer cells.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2023Article
- Modeling Duchenne Muscular Dystrophy Cardiomyopathy with Patients' Induced Pluripotent Stem-Cell-Derived Cardiomyocytes.International journal of molecular sciences · 2023Review
- Full-length human dystrophin on human artificial chromosome compensates for mouse dystrophin deficiency in a Duchenne muscular dystrophy mouse model.Scientific reports · 2023Article
- Defining the Skeletal Myogenic Lineage in Human Pluripotent Stem Cell-Derived Teratomas.Cells · 2022Article
- Perspectives on hiPSC-Derived Muscle Cells as Drug Discovery Models for Muscular Dystrophies.International journal of molecular sciences · 2021Review
- Highly Efficient Microcell-Mediated Transfer of HACs Containing a Genomic Region of Interest into Mammalian Cells.Current protocols · 2021Article
- Article
- A novel DMD intronic alteration: a potentially disease-causing variant of an intermediate muscular dystrophy phenotype.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2021Article
- Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling.Molecular therapy. Nucleic acids · 2021Article
- Pax7 as molecular switch regulating early and advanced stages of myogenic mouse ESC differentiation in teratomas.Stem cell research & therapy · 2020Article
- Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders.Molecular therapy. Methods & clinical development · 2020Article
- Human AlphoidCells · 2020Article
- Harnessing the Potential of Stem Cells for Disease Modeling: Progress and Promises.Journal of personalized medicine · 2020Review
- Studying Abnormal Chromosomal Diseases Using Patient-Derived Induced Pluripotent Stem Cells.Frontiers in cellular neuroscience · 2020Article
- Application of iPSC to Modelling of Respiratory Diseases.Advances in experimental medicine and biology · 2020Review
59 more citing papers are in PubMed but not listed here.
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Authors and funding
16 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Human artificial chromosome (HAC) has several advantages as a gene therapy vector, including stable episomal maintenance that avoids insertional mutations and the ability to carry large gene inserts including the regulatory elements. Induced pluripotent stem (iPS) cells have great potential for gene therapy, as such cells can be generated from the individual's own tissues, and when reintroduced can contribute to the specialized function of any tissue. As a proof of concept, we show herein the complete correction of a genetic deficiency in iPS cells derived from Duchenne muscular dystrophy (DMD) model (mdx) mice and a human DMD patient using a HAC with a complete genomic dystrophin sequence (DYS-HAC). Deletion or mutation of dystrophin in iPS cells was corrected by transferring the DYS-HAC via microcell-mediated chromosome transfer (MMCT). DMD patient- and mdx-specific iPS cells with the DYS-HAC gave rise to differentiation of three germ layers in the teratoma, and human dystrophin expression was detected in muscle-like tissues. Furthermore, chimeric mice from mdx-iPS (DYS-HAC) cells were produced and DYS-HAC was detected in all tissues examined, with tissue-specific expression of dystrophin. Therefore, the combination of patient-specific iPS cells and HAC-containing defective genes represents a powerful tool for gene and cell therapies.
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