Project: Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors
PI:
Erik J Sontheimer, PhD
Summary of data submissions:
- Data for 10 experiments were submitted on 2021-04-15 SCGE ID:1030
- Data for 1 experiments were submitted on 2021-10-01 SCGE ID:1052
Submissions Details
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| Experiment Name | Type | Description |
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Testing newly chemically modified crRNA and tracrRNA in mTmG mouse embryonic fibroblasts |
In Vitro | Chemically modified crRNA and tracrRNA were delivered by electroporation to embryonic fibroblasts harvested from the mTmG reporter mouse. Gene editing was determined by reporter activation. |
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Testing newly chemically modified crRNA and tracrRNA in mouse Hepa 1-6 cells |
In Vitro | Chemically modified crRNA and tracrRNA were delivered by electroporation to mouse Hepa 1-6 cells. Editing activity was determined by Sanger sequencing |
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Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain |
In Vivo | Chemically modified crRNA and tracrRNA were injected into the intra-striatum of mTmG reporter mice and activation of GFP expression was imaged. |
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Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain |
In Vivo | Chemically modified crRNA and tracrRNA were injected into the intra-striatum of mTmG reporter mice and activation of GFP expression was imaged. |
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Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain |
In Vivo | Chemically modified crRNA and tracrRNA were injected into the intra-striatum of mTmG reporter mice and activation of GFP expression was imaged. |
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Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain |
In Vivo | Chemically modified crRNA and tracrRNA were injected into the intra-striatum of mTmG reporter mice and activation of GFP expression was imaged. |
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Testing newly chemically modified crRNA and tracrRNA to activate the TLR reporter in human cells |
In Vitro | Chemically modified crRNA and tracrRNA were delivered by electroporation in presence of amphiphilic peptide to transgenic human HEK-293T cells harboring the TLR-MCV1 reporter. Gene editing was determined by reporter activation. |
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Testing newly chemically modified crRNA and tracrRNA to activate the TLR1 reporter in human cells |
In Vitro | Chemically modified crRNA and tracrRNA were delivered by electroporation to transgenic human HEK-293T cells harboring the TLR1 reporter. Gene editing was determined by reporter activation. |
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Testing newly chemically modified crRNA and tracrRNA in mouse Neuro 2A cells |
In Vitro | Chemically modified crRNA and tracrRNA were delivered by electroporation to mouse Neuro2A cells. Editing activity was determined by Sanger sequencing |
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Delivery of RNP containing chemically modified crRNA C20 with chemically modified tracrRNA T2-PS to determine the RNP distribution in TLR-MCV mouse brain |
In Vivo | Chemically modified crRNA and tracrRNA were injected into the striatum of TLR-MCV mice and the distribution of RNP was imaged. |
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| Experiment Name | Type | Description | |||
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Testing preparation for independent validation at The Jackson Laboratory Small Animal Testing Center |
In Vivo | Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain | |||
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