Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9
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Summary
Adenoviral-mediated delivery of CRISPR/Cas9 achieved gene editing outcomes persisting over 200 days and gene knock-in maintained greater levels of the serum protein than provided by episomal expression, which is generalizable to other serum proteins and supports in vivo cDNA replacement therapy to achieve stable gene expression.
- Type
- article
- Published
- 2018-03-27
- Cited by
- 48
- References
- 74
- Access
- Open access
- OpenAlex
- https://openalex.org/W2796001122
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:4420063
Keywords
CRISPR, Biology, Cas9, Gene knockin, Genetic enhancement
References
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- Biosynthesis of human von Willebrand factor.
- Hepatic Gene Therapy: Persistent Expression of Human α1-Antitrypsin in Mice after Direct Gene Delivery In Vivo
- A Comparison of Exogenous Promoter Activity at the ROSA26 Locus Using a PhiC31 Integrase Mediated Cassette Exchange Approach in Mouse ES Cells
- Genomic cis-acting Sequences Improve Expression and Establishment of a Nonviral Vector
- Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9
- Efficient gene targeting of the Rosa26 locus in mouse zygotes using TALE nucleases
- Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo.
Cited by
- Applications of CRISPR systems in respiratory health: Entering a new ‘red pen’ era in genome editing
- Nanoparticle based delivery of CRISPR/Cas9 genome editing therapeutics
- Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9
- Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases
- Recent advances in CRISPR/Cas9-mediated knock-ins in mammalian cells.
- Genome editing technologies to treat rare liver diseases.
- An efficient gene knock-in strategy using 5’-modified dsDNA donors with short homology arms
- A New Gorilla Adenoviral Vector with Natural Lung Tropism Avoids Liver Toxicity and Is Amenable to Capsid Engineering and Vector Retargeting
- Gene editing and CRISPR in the clinic: current and future perspectives
- The delivery challenge: fulfilling the promise of therapeutic genome editing
- Advances in Alpha-1 Antitrypsin Gene Therapy.
- The Evolution of Gene Therapy in the Treatment of Metabolic Liver Diseases
- Gene Therapy in Rare Respiratory Diseases: What Have We Learned So Far?
- New Directions in Pulmonary Gene Therapy
- Adenoviral Vectors for In Vivo Delivery of CRISPR-Cas Gene Editors
- Liver targeted gene therapy: Insights into emerging therapies.
- Alpha-1 antitrypsin deficiency and recombinant protein sources with focus on plant sources: updates, challenges and perspectives.
- Efficient viral delivery of Cas9 into human safe harbor
- Targeted Gene Delivery: Where to Land
- Genome editing in the human liver: Progress and translational considerations.
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