ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017
Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed, 34 citations in OpenAlex.
- Bright Ferritin for Non-Invasive MRI Monitoring of the Fate of Transplanted hPSC-Cardiomyocytes in the Infarcted Rat Heart.Magnetic resonance in medicine · 2026Article
- Engineering of rhesus monkey pluripotent stem cells for noninvasive survey and remote control after brain allotransplantation.Fundamental research · 2026Article
- Interspecies Organoids Reveal Human-Specific Molecular Features of Dopaminergic Neuron Development and Vulnerability.bioRxiv : the preprint server for biology · 2025Article
- Synergy between pluripotent stem cell-derived macrophages and self-renewing macrophages: Envisioning a promising avenue for the modelling and cell therapy of infectious diseases.Cell proliferation · 2025Review
- Effective and stable gene transduction in rhesus macaque iPSCs capable of T-lineage differentiation utilizing the piggyBac system.Regenerative therapy · 2024Article
- Long-term engraftment and maturation of autologous iPSC-derived cardiomyocytes in two rhesus macaques.Cell stem cell · 2024Article
- Generation and characterization of inducible KRAB-dCas9 iPSCs from primates for cross-species CRISPRi.iScience · 2024Article
- An expedition in the jungle of pluripotent stem cells of non-human primates.Stem cell reports · 2023Review
- Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells.Stem cell reviews and reports · 2023Review
- Seeing Neurodegeneration in a New Light Using Genetically Encoded Fluorescent Biosensors and iPSCs.International journal of molecular sciences · 2023Review
- The sound of silence: Transgene silencing in mammalian cell engineering.Cell systems · 2022Review
- Article
- Addressing the dark matter of gene therapy: technical and ethical barriers to clinical application.Human genetics · 2022Review
- Retinal organoids derived from rhesus macaque iPSCs undergo accelerated differentiation compared to human stem cells.Cell proliferation · 2022Article
- Discovery and validation of human genomic safe harbor sites for gene and cell therapies.Cell reports methods · 2022Article
- Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- A piggyBac-based platform for genome editing and clonal rhesus macaque iPSC line derivation.Scientific reports · 2021Article
- Generation of macrophages with altered viral sensitivity from genome-edited rhesus macaque iPSCs to model human disease.Molecular therapy. Methods & clinical development · 2021Article
- CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside.British journal of haematology · 2021Review
- CRISPR/Cas9-mediated introduction of the sodium/iodide symporter gene enables noninvasive in vivo tracking of induced pluripotent stem cell-derived cardiomyocytes.Stem cells translational medicine · 2020Article
Corrections and comments
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Authors and funding
21 authors at 6 institutions in 2 countries.
Funding
Abstract
Nonhuman primate (NHP) induced pluripotent stem cells (iPSCs) offer the opportunity to investigate the safety, feasibility, and efficacy of proposed iPSC-derived cellular delivery in clinically relevant in vivo models. However, there is need for stable, robust, and safe labeling methods for NHP iPSCs and their differentiated lineages to study survival, proliferation, tissue integration, and biodistribution following transplantation. Here we investigate the utility of the adeno-associated virus integration site 1 (AAVS1) as a safe harbor for the addition of transgenes in our rhesus macaque iPSC (RhiPSC) model. A clinically relevant marker gene, human truncated CD19 (hΔCD19), or GFP was inserted into the AAVS1 site in RhiPSCs using the CRISPR/Cas9 system. Genetically modified RhiPSCs maintained normal karyotype and pluripotency, and these clones were able to further differentiate into all three germ layers in vitro and in vivo. In contrast to transgene delivery using randomly integrating viral vectors, AAVS1 targeting allowed stable transgene expression following differentiation. Off-target mutations were observed in some edited clones, highlighting the importance of careful characterization of these cells prior to downstream applications. Genetically marked RhiPSCs will be useful to further advance clinically relevant models for iPSC-based cell therapies.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.