ArticleMolecular therapy. Methods & clinical development2020
Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders.
Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 19 citations in OpenAlex.
- Case report: Whole exome sequencing identifies a novel variant in theFrontiers in genetics · 2025Article
- Microcell-mediated chromosome transfer between non-identical human iPSCs.Molecular therapy. Nucleic acids · 2024Article
- ChromTR: chromosome detection in raw metaphase cell images via deformable transformers.Frontiers of medicine · 2024Article
- Review
- Rapid human genomic DNA cloning into mouse artificial chromosome via direct chromosome transfer from human iPSC and CRISPR/Cas9-mediated translocation.Nucleic acids research · 2024Article
- Treatment of CHO cells with Taxol and reversine improves micronucleation and microcell-mediated chromosome transfer efficiency.Molecular therapy. Nucleic acids · 2023Article
- First sex modification case in equine cloning.PloS one · 2023Article
- Dysfunctional Extracellular Matrix Remodeling Supports Perianal Fistulizing Crohn's Disease by a Mechanoregulated Activation of the Epithelial-to-Mesenchymal Transition.Cellular and molecular gastroenterology and hepatology · 2023Article
- Review
- Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling.Molecular therapy. Nucleic acids · 2021Article
- Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes.Cellular and molecular life sciences : CMLS · 2021Review
- Generation of Induced Pluripotent Stem Cells from a Female Patient with a Xq27.3-q28 Deletion to Establish Disease Models and Identify Therapies.Cellular reprogramming · 2020Article
Corrections and comments
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Authors and funding
13 authors at 5 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Many human genetic diseases are associated with gross mutations such as aneuploidies, deletions, duplications, or inversions. For these "structural" disorders, conventional gene therapy, based on viral vectors and/or on programmable nuclease-mediated homologous recombination, is still unsatisfactory. To correct such disorders, chromosome transplantation (CT), defined as the perfect substitution of an endogenous defective chromosome with an exogenous normal one, could be applied. CT re-establishes a normal diploid cell, leaving no marker of the procedure, as we have recently shown in mouse pluripotent stem cells. To prove the feasibility of the CT approach in human cells, we used human induced pluripotent stem cells (hiPSCs) reprogrammed from Lesch-Nyhan (LN) disease patients, taking advantage of their mutation in the X-linked
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Registered trials
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