ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Engineering of human pluripotent stem cells by AAV-mediated gene targeting.
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 77 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
77 citing papers in PubMed, 112 citations in OpenAlex.
- AAV Vectors in Regenerative Medicine and Cellular Reprogramming: Potential, Pitfalls, and Specificity Constraints.International journal of molecular sciences · 2026Review
- Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus.Human gene therapy · 2024Review
- Structural basis for nuclear import of bat adeno-associated virus capsid protein.The Journal of general virology · 2024Article
- "Cutting the Mustard" with Induced Pluripotent Stem Cells: An Overview and Applications in Healthcare Paradigm.Stem cell reviews and reports · 2022Review
- Developing Bottom-Up Induced Pluripotent Stem Cell Derived Solid Tumor Models Using Precision Genome Editing Technologies.The CRISPR journal · 2022Review
- Inborn errors of metabolism: Lessons from iPSC models.Reviews in endocrine & metabolic disorders · 2021Review
- The Potential of Induced Pluripotent Stem Cells to Test Gene Therapy Approaches for Neuromuscular and Motor Neuron Disorders.Frontiers in cell and developmental biology · 2021Review
- Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?Frontiers in immunology · 2021Review
- Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies.Advances in experimental medicine and biology · 2020Review
- One Step Into the Future: New iPSC Tools to Advance Research in Parkinson's Disease and Neurological Disorders.Journal of Parkinson's disease · 2019Review
- Nuclease-free Adeno-Associated Virus-Mediated Il2rg Gene Editing in X-SCID Mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia.Arteriosclerosis, thrombosis, and vascular biology · 2018Review
- Comparative analysis of chimeric ZFP-, TALE- and Cas9-piggyBac transposases for integration into a single locus in human cells.Nucleic acids research · 2017Article
- HLA-E-expressing pluripotent stem cells escape allogeneic responses and lysis by NK cells.Nature biotechnology · 2017Article
- Adeno-Associated Virus Vectors and Stem Cells: Friends or Foes?Human gene therapy · 2017Review
- Integration-defective lentiviral vector mediates efficient gene editing through homology-directed repair in human embryonic stem cells.Nucleic acids research · 2017Article
- Genome Editing for the Study of Cardiovascular Diseases.Current cardiology reports · 2017Review
- Review
- Nanotechnology for delivery of peptide nucleic acids (PNAs).Journal of controlled release : official journal of the Controlled Release Society · 2016Review
- Genome engineering tools for building cellular models of disease.The FEBS journal · 2016Review
17 more citing papers are in PubMed but not listed here.
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Authors and funding
10 authors at 1 institution in 1 country.
Funding
Abstract
Precise genetic manipulation of human pluripotent stem cells will be required to realize their scientific and therapeutic potential. Here, we show that adeno-associated virus (AAV) gene targeting vectors can be used to genetically engineer human embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs). Different types of sequence-specific changes, including the creation and correction of mutations, were introduced into the human HPRT1 and HMGA1 genes (HPRT1 mutations being responsible for Lesch-Nyhan syndrome). Gene targeting occurred at high frequencies in both ESCs and iPSCs, with over 1% of all colony-forming units (CFUs) undergoing targeting in some experiments. AAV vectors could also be used to target genes in human fibroblasts that were subsequently used to derive iPSCs. Accurate and efficient targeting took place with minimal or no cytotoxicity, and most of the gene-targeted stem cells produced were euploid and pluripotent.
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What OpenQuestion holds
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.