ArticleMolecular therapy : the journal of the American Society of Gene Therapy2016
High Efficiency CRISPR/Cas9-mediated Gene Editing in Primary Human T-cells Using Mutant Adenoviral E4orf6/E1b55k "Helper" Proteins.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 35 citations in OpenAlex.
- Viral genome editing methods and applications in the CRISPR era.Journal of virology · 2026Review
- Fine-Tuning Homology-Directed Repair (HDR) for Precision Genome Editing: Current Strategies and Future Directions.International journal of molecular sciences · 2025Review
- Delivering CRISPR to the HIV-1 reservoirs.Frontiers in microbiology · 2024Review
- Current Applications and Future Perspectives of CRISPR-Cas9 for the Treatment of Lung Cancer.Biologics : targets & therapy · 2021Review
- CRISPR-Based Editing Techniques for Genetic Manipulation of Primary T Cells.Methods and protocols · 2020Review
- Advances in Alpha-1 Antitrypsin Gene Therapy.American journal of respiratory cell and molecular biology · 2020Review
- Genome-edited adult stem cells: Next-generation advanced therapy medicinal products.Stem cells translational medicine · 2020Review
- Efficient CRISPR/Cas9 Disruption of Autoimmune-Associated Genes Reveals Key Signaling Programs in Primary Human T Cells.Journal of immunology (Baltimore, Md. : 1950) · 2019Article
- Gene editing: Towards the third generation of adoptive T-cell transfer therapies.Immuno-oncology technology · 2019Review
- Cancer diagnosis and immunotherapy in the age of CRISPR.Genes, chromosomes & cancer · 2019Review
- Application of Genome Editing Techniques in Immunology.Archivum immunologiae et therapiae experimentalis · 2018Review
- Synthetic biology advances and applications in the biotechnology industry: a perspective.Journal of industrial microbiology & biotechnology · 2018Article
- Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome.Cell reports · 2018Article
- Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9.Gene therapy · 2018Article
- CRISPR/Cas9 Technology as an Emerging Tool for Targeting Amyotrophic Lateral Sclerosis (ALS).International journal of molecular sciences · 2018Review
- Engineering Protein-Secreting Plasma Cells by Homology-Directed Repair in Primary Human B Cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Targeted Delivery of CRISPR/Cas9-Mediated Cancer Gene Therapy via Liposome-Templated Hydrogel Nanoparticles.Advanced functional materials · 2017Article
- Homology-Directed Recombination for Enhanced Engineering of Chimeric Antigen Receptor T Cells.Molecular therapy. Methods & clinical development · 2017Article
- Method for Dual Viral Vector Mediated CRISPR-Cas9 Gene Disruption in Primary Human Endothelial Cells.Scientific reports · 2017Article
- Review
Corrections and comments
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Authors and funding
13 authors at 2 institutions in 1 country.
Funding
Abstract
Many future therapeutic applications of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 and related RNA-guided nucleases are likely to require their use to promote gene targeting, thus necessitating development of methods that provide for delivery of three components-Cas9, guide RNAs and recombination templates-to primary cells rendered proficient for homology-directed repair. Here, we demonstrate an electroporation/transduction codelivery method that utilizes mRNA to express both Cas9 and mutant adenoviral E4orf6 and E1b55k helper proteins in association with adeno-associated virus (AAV) vectors expressing guide RNAs and recombination templates. By transiently enhancing target cell permissiveness to AAV transduction and gene editing efficiency, this novel approach promotes efficient gene disruption and/or gene targeting at multiple loci in primary human T-cells, illustrating its broad potential for application in translational gene editing.
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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.