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Making genome editing delivery vehicles in the body to amplify editing efficiencies

Award Number: K99EB039138
ORGANIZATION: NATIONAL INSTITUTE Of BIOMEDICAL IMAGING & BIOENGINEERING
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: TRAINING/TRAINEESHIP
PERIOD OF PERFORMANCE START DATE: 05/01/2026
PERIOD OF PERFORMANCE END DATE: 04/30/2028

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Making genome editing delivery vehicles in the body to amplify editing efficiencies - 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.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $120,393 )
2026	2026	REGENTS OF THE UNIVERSITY OF CALIFORNIA, THE	119 CALIFORNIA HALL	BERKELEY	CA	94720	ALAMEDA	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	000	1	4/27/2026	NEW	$120,393
														Subtotal = $120,393

Grand Total All Awards = $120,393

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Making genome editing delivery vehicles in the body to amplify editing efficiencies

Award Number: K99EB039138

ORGANIZATION: NATIONAL INSTITUTE Of BIOMEDICAL IMAGING & BIOENGINEERING

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: TRAINING/TRAINEESHIP

PERIOD OF PERFORMANCE START DATE: 05/01/2026

PERIOD OF PERFORMANCE END DATE: 04/30/2028

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