Making genome editing delivery vehicles in the body to amplify editing efficiencies
NIBIB - National Institute of Biomedical Imaging and Bioengineering
About This Grant
PROJECT SUMMARY/ABSTRACT Directly genome editing cells inside the body could treat numerous genetic diseases, including sickle cell disease. However, genome editing of most cell types outside of the liver, such as hematopoietic stem cells, is limited by inefficient delivery. Only a small subset of cells inside of a tissue are accessible to delivery vehicles and editing enzymes. To overcome this delivery problem, my central objective is to enable cells initially receiving delivery vehicles to transiently produce and distribute genome editing enzymes to neighboring cells in vesicles. This allows editing activity to spread beyond initial delivery. My proposal builds on my postdoctoral research studying the delivery mechanisms of Enveloped Delivery Vehicles (EDVs). EDVs are lentivirus-derived lipid vesicles engineered to package CRISPR-Cas9 ribonucleoproteins that can be targeted to specific cell types using surface-displayed fusogens and antibodies. I hypothesize that transient, local production of EDVs in vivo will amplify genome editing efficiency by increasing the concentration of genome editing enzymes and enabling their spread across cells. This hypothesis will be tested through three specific aims: (1) develop single nucleic acid molecules encoding EDVs, (2) establish methods to target EDV production and uptake to specific cell types, and (3) deliver EDV-encoding plasmids to amplify editing in vivo. In preliminary work for this proposal, I showed that hydrodynamic injection of EDV-encoding plasmids into mice amplified genome editing efficiencies compared to Cas9 only plasmid controls. Successful completion of this proposal will generate fundamental insights into propagating genome editing effects beyond cells initially reached by delivery vehicles starting with hematopoietic stem cells as a model therapeutic cell type. This approach could broadly transform biological therapy delivery by overcoming low delivery efficiencies through localized amplification and spread of therapeutic macromolecules. The training acquired through this proposal in primary cell culture, bioinformatics, and next-generation sequencing will bolster my readiness to lead an independent research program. I will be mentored by Dr. Jennifer Doudna, a global leader in genome editing technology, and leading experts and clinicians in primary cell engineering, hematopoietic stem cell biology and virology in the California scientific community. During the mentored phase of this project, I will hone my scientific and professional skills to become a scientific leader. I will engage in structured professional development activities and actively present my research at leading conferences to facilitate a successful transition to an independent academic research position. The vibrant and collaborative environment provided by UC Berkeley and the Innovative Genomics Institute offers an outstanding environment to complete my training and start my independent scientific career.
Grant Summary
Making genome editing delivery vehicles in the body to amplify editing efficiencies is a NIBIB - National Institute of Biomedical Imaging and Bioengineering grant providing up to $120K for university, nonprofit, healthcare org. Applications are due 2028-04-30 (open). Check eligibility and apply with FindGrants.
Focus Areas
Eligibility
How to Apply
Up to $120K
2028-04-30
- 1Confirm your organization is eligible for Making genome editing delivery vehicles in the body to amplify editing efficiencies from NIBIB - National Institute of Biomedical Imaging and Bioengineering, checking organization type, location, and any population or project requirements.
- 2Gather the required documents and information, including your organization details, project plan, and budget figures.
- 3Draft your application narrative and budget addressing the funder's priorities and review criteria. FindGrants can draft each section for you to review and edit.
- 4Review every section against the requirements checklist, then export a submission-ready application pack and submit it to NIBIB - National Institute of Biomedical Imaging and Bioengineering before the deadline.
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Making genome editing delivery vehicles in the body to amplify editing efficiencies: Frequently Asked Questions
Who is eligible for the Making genome editing delivery vehicles in the body to amplify editing efficiencies?
Making genome editing delivery vehicles in the body to amplify editing efficiencies is offered by NIBIB - National Institute of Biomedical Imaging and Bioengineering and is generally open to university, nonprofit, healthcare org. It is open to organizations nationwide unless the funder specifies otherwise. Review the specific eligibility terms before applying, since funders set their own requirements around organization type, location, and the population or project being served.
How much funding does the Making genome editing delivery vehicles in the body to amplify editing efficiencies provide?
Making genome editing delivery vehicles in the body to amplify editing efficiencies provides up to $120K per award from NIBIB - National Institute of Biomedical Imaging and Bioengineering. Actual award sizes depend on the scope of your project, available program funds, and the number of applicants, so build a budget that reflects realistic, allowable costs rather than the maximum figure.
When is the Making genome editing delivery vehicles in the body to amplify editing efficiencies deadline?
Applications for Making genome editing delivery vehicles in the body to amplify editing efficiencies are due 2028-04-30 (open). Because deadlines can change, verify the date with the funder, NIBIB - National Institute of Biomedical Imaging and Bioengineering, and give yourself enough time to prepare a complete, competitive application before the close date.
How do you apply for the Making genome editing delivery vehicles in the body to amplify editing efficiencies?
To apply for Making genome editing delivery vehicles in the body to amplify editing efficiencies, confirm your eligibility, gather the required documents, and prepare a narrative and budget that address the funder's priorities. FindGrants guides you step by step and can draft each section, then exports a submission-ready application pack for this grant from NIBIB - National Institute of Biomedical Imaging and Bioengineering.