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 #
1R44HD090831-01
Application #
9255240
Study Section
Special Emphasis Panel (ZRG1-ETTN-L (11)F)
Program Officer
Mukhopadhyay, Mahua
Project Start
2016-09-23
Project End
2017-08-31
Budget Start
2016-09-23
Budget End
2017-08-31
Support Year
1
Fiscal Year
2016
Total Cost
$225,000
Indirect Cost

  Institution

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