Feasibility of inspire technology to enhance crispr mediated genome editors in vivo
Ended Project Grant Awarded Last action
- Award ID
- R43GM143999
- Recipient
-
Gradient Medical Inc. UEI HPZ7FS9MF8J1
- Awarding agency
-
National Institute of General Medical Sciences
National Institutes of Health, Department of Health and Human Services
- Obligated
- $755,516
Award facts
- Assistance listing
- 93.859 Biomedical Research and Research Training
- Completion date
- Originally
- Funding
-
- $503,602.00 Federal
- $251,914.00 Non-federal
- Place of performance
- North Carolina, USA
Award details come from USAspending.gov.
Description
- Abstract
- This applications long-term objective is to develop a platform technology enabling in vivo genetic engineering technologies including DNA vaccination and precision crispr gene editing. A challenge in utilizing crispr technologies in vivo is the need to simultaneously deliver the DNA endonuclease enzyme, guide RNA, and payload DNA. Integrated nanosecond pulse intradermal reversible electroporation (inspire) is a novel technique for enhancing in vivo delivery of these components. Inspire rapidly transports large molecules into cells by using ultrashort electrical pulses to simultaneously induce temporary nanoscale defects in the cell membrane and drive the target molecules against concentration gradients via electrokinetic drift. This proposal will demonstrate the feasibility of inspire for in vivo genetic engineering in a 3D in vitro skin model and a porcine dermal model via three specific aims. 1) development of a hand held system suitable for clinical inspire treatments, 2) optimization of inspire protocols in a laboratory model, and 3) feasibility of inspire protocols in a large animal model. In aim 1 we will construct a portable pulse generation system suitable for clinical use with the high voltage components integrated directly into a hand held applicator. In aim 2 we will optimize protocols for delivering crispr CAS9 plasmid, CAS9 mrna, and CAS9 protein into cells using a 3D in vitro skin mimic. In aim 3 we will demonstrate the feasibility of delivering these crispr components in vivo via a porcine dermal model. Following this phase i application demonstrating in vivo feasibility, phase II will focus on design for manufacture and initiate long-term safety/efficacy studies designed to determine serum levels of antibodies produced following transfection with DNA vaccine candidates.
Transactions
| Action | Federal | Non-federal | Date |
|---|---|---|---|
| Amendment | -$226 | $0 | |
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| Amendment | $0 | $0 | |
| Amendment | $0 | $0 | |
| Amendment | $0 | $0 | |
| Amendment | $0 | $0 | |
| Amendment | $0 | $0 | |
| Amendment | $251,914 | $0 | |
| Original award | $251,914 | $251,914 |
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Quick answers
Who received award R43GM143999?
R43GM143999 was awarded to Gradient Medical Inc. by National Institute of General Medical Sciences (National Institutes of Health) on September 1, 2021.
How much is award R43GM143999 worth?
$755,516 has been obligated on R43GM143999: $503,602 in federal funding and $251,914 in non-federal funding.
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