CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.
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Summary
The rapidly developing CRISPR/Cas9-based technologies for disease modeling and gene correction and recent progress toward Cas9/guide RNA (gRNA) delivery based on viral and nonviral vectors are reviewed.
- Type
- review
- Published
- 2017-06-22
- Cited by
- 489
- References
- 299
- OpenAlex
- https://openalex.org/W2667943050
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:8728254
Keywords
CRISPR, Genome editing, Cas9, Guide RNA, Computational biology
References
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- Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals
- A peptide nucleic acid–nuclear localization signal fusion that mediates nuclear transport of DNA
- Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing
- Genetic screens in human cells using the CRISPR/Cas9 system
- Development of an intein-mediated split–Cas9 system for gene therapy
- In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency
- Cas9 gRNA engineering for genome editing, activation and repression
- Genome engineering using CRISPR-Cas9 system.
- DNA-free genome editing in plants with preassembled CRISPR-Cas9 ribonucleoproteins
Cited by
- Methods and applications of CRISPR-mediated base editing in eukaryotic genomes
- Nanoparticles for CRISPR–Cas9 delivery
- CRISPR-Based Antibacterials: Transforming Bacterial Defense into Offense.
- Recent Advances in Nucleic Acid Targeting Probes and Supramolecular Constructs Based on Pyrene-Modified Oligonucleotides
- CRISPR genome editing and its medical applications
- Endosomal Escape and Delivery of CRISPR/Cas9 Genome Editing Machinery Enabled by Nanoscale Zeolitic Imidazolate Framework.
- The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
- The physicist’s guide to one of biotechnology’s hottest new topics: CRISPR-Cas
- A beginner's guide to gene editing
- Engineering the Delivery System for CRISPR-based Genome Editing
- Therapeutic Peptide Nucleic Acids: Principles, Limitations, and Opportunities
- Combinatorial library of chalcogen-containing lipidoids for intracellular delivery of genome-editing proteins.
- High-performance cationic polyrotaxanes terminated with polypeptides as promising nucleic acid delivery systems
- Tapping the RNA world for therapeutics
- Optimization of lipid-assisted nanoparticle for disturbing neutrophils-related inflammation.
- Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing.
- Efficient CRISPR‐based genome editing using tandem guide RNAs and editable surrogate reporters
- Systemic delivery of CRISPR/Cas9 with PEG-PLGA nanoparticles for chronic myeloid leukemia targeted therapy.
- Nanoscale platforms for messenger RNA delivery
- Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities
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