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6 results for search term 'CRISPR' in category Project
Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic PeptidesProject [Delivery Systems]![]() Show Experiments (6)
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Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung DiseasesProject [Delivery Systems]![]() |
Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the BrainProject [Delivery Systems]![]() |
Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and DonorsProject [Delivery Systems]![]() Show Experiments (11)
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Expanding CRISPR-Cas Editing Technology through Exploration of Novel Cas Proteins and DNA Repair SystemsProject [Genome Editors]![]() Show Experiments (1) |
Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative DisordersProject [Delivery Systems]![]() Show Experiments (1) |
6 results for search term 'CRISPR' in category Project
Type | Subtype | Name | Description | Source | Last Updated Date | View Associated... |
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Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides | The proposed research is relevant to the public health because genetic and acquired diseases affecting the airways pose major disease and economic burdens. By advancing the delivery of gene editing tools, it may be possible to therapeutically modify the cells lining the airways. This novel strategy has implications for the treatment of both monogenetic and acquired lungs disease, and may have applications for other somatic cell therapies. | 2020-11-02 |
Show Experiments (6)
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Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases | Efficacy and safety limitations in current gene editing technologies have hindered efforts to treat genetic lung diseases. This proposal seeks to develop and validate a combinatorial delivery approach that uses non-viral and viral vehicles to efficiently transport gene editing tools to disease-relevant cells in the lung. Completion of our work will establish safe and effective delivery vehicles that will guide the design of future gene therapies for genetic disorders. | 2020-10-20 | ||||
Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain | In vivo genome editing using CRISPR/Cas9 is anticipated to be the next wave of therapeutics for various major health threats, including neurodegenerative diseases. However, to date, very few Cas9-gRNA ribonucleoprotein in vivo delivery methods have been reported, and delivery to the brain has been particularly challenging. The unique nanocapsules we plan to develop will ultimately enable high efficiency neuron-targeted genome editing in the brain, thereby offering new hope to treat devastating neurodegenerative diseases. | 2020-10-28 | ||||
Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors | RNA-guided CRISPR genome editing systems promise to revolutionize the treatment of inherited disease. Safe, effective, and target-tissue-specific delivery of the guide RNA that directs editing is a critical hurdle in the development of clinical applications for engineered CRISPR systems. Using strategies validated for the delivery of other categories of nucleic acid therapeutics, we have established a framework for complete chemical modification of CRISPR guides, thereby conferring in vivo stability and effective biodistribution properties. The proposed research will optimize these guides, as well as other editing components, for clinical use. | 2021-04-15 |
Show Experiments (11)
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Expanding CRISPR-Cas Editing Technology through Exploration of Novel Cas Proteins and DNA Repair Systems | Expanding genome editing tools through exploration of new CRISPR-Cas proteins and DNA repair enzymes NARRATIVE Fundamental research on bacterial adaptive immunity uncovered the genome editing properties of CRISPR-Cas systems, and it is clear that uncultivated microbes contain more pathways and enzymes that may be useful as genome editing tools. We will combine bioinformatics and biochemistry to identify new DNA- and RNA-associating proteins and will analyze their mechanisms of action. We will focus our investigation on newly described CRISPR-Cas systems and DNA-interacting proteins that occur in conserved genomic context. | 2020-10-16 |
Show Experiments (1) |
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Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders | Gene editing may offer a new therapeutic strategy to tackle many neurodegenerative disorders that remain untreatable. Current methodologies of delivering CRISPR-based gene editing elements to the brain are highly inefficient. We propose to develop a noninvasive, efficient approach to achieve gene editing in the brain using focused ultrasound technology. | 2022-04-15 |
Show Experiments (1) |