Abstract

Thebroad,longtermobjectiveandspecificaimsofthefasttrackSBIRresearcharetocreatea reagent,RecombiRED,foruseinmicroinjectionofembryosforidentifyingasubsetofembryos asrecombinationcompetentinjections.Theabilitytoquicklyidentifygenomeeditingeventsin anembryoallowsresearchertoquicklymakeprecisionanimalmodelsofhumandiseasestates. Preciseanimalmodelsallowforgreaterscientificunderstandingofahumandiseaseand providesarobustplatformfordrugdiscoveryscreening.TheRecombiREDreagentrelieson inducedfluorescenceinresponsetoactivationofhomologousrecombinationrepairwithinthe embryo.DetectionofhomologousrecombinationrepairininjectedembryoswithRecombiRED allowsanimalhusbandryandfurtherscreeningeffortstoberestrictedtoasubsetofembryosby upto100xforhavingadesiredpreciseknockinofnewgeneticmaterialataspecificgenetic locus.ThephaseIresearchadaptsatheRecombiREDreagentdevelopedinC .elegansmodel organismtohavetheappropriateexpressioncontrolelementsenablingsufficientactivityin zebrafishembryo.InPhaseII,theadditionalresearchfurtheroptimizesparametersofthe RecombiREDreagenttominimizefalsepositiveandfalsenegativereadouts,whichthenallows maximalforecastingoftargetedgenomeeditevents.Finally,asetof15realworldtransgenesis projectsareusedaspremarketbetatesttodemonstratetheubiquityandrobustnessofthe RecombiREDforsimplifyingdiscoveryofgenomeeditinginthecreationofanimalsmodelsfor useindrugdiscovery.

Public Health Relevance

ofthisresearchtopublichealthisthecreationofanexperimentaltoolfor simplifyingthediscoveryofgeneticallyalteredmodelorganisms.Thetoolallowsfastercreation ofanimalmodelsforuseinbetterscientificdiscoveryandfasterdrugdevelopment.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Small Business Innovation Research Grants (SBIR) - Phase II (R44)
Project #
5R44HD090831-03
Application #
9520355
Study Section
Special Emphasis Panel (ZRG1)
Program Officer
Mukhopadhyay, Mahua
Project Start
2017-07-01
Project End
2019-11-30
Budget Start
2018-12-01
Budget End
2019-11-30
Support Year
3
Fiscal Year
2019
Total Cost
Indirect Cost

  Institution

Name
Kdt, Inc
Department
Type
DUNS #
832418342
City
Murray
State
UT
Country
United States
Zip Code
84123