2022-01-31Orphan Disease CenterVivian, Jay2021-02-01Platforms for the identification and validation of candidate small molecule treatments for TBCK syndromeMDBR-21-109-TBCKcmhprofilesont:personsuffixperson suffixprns:endDateend dateFaculty Rankprns:fullNamefull nameprns:grantAwardedBygrant awarded byprns:hasFacultyRankhas faculty rankprns:isPrimaryPositionis primary positionprns:latitudelatitudeprns:longitudelongitudeprns:mainImagephotoprns:personIdPerson IDprns:personInPrimaryPositionperson in primary positionprns:positionInDepartmentposition in departmentprns:positionInDivisionposition in divisionprns:principalInvestigatorNameprincipal investigator nameprns:sortOrdersort orderprns:startDatestart dateAgreementvivo:contributingRolecontributorDepartmentvivo:descriptiondescriptionDivisionGrantvivo:hrJobTitleHR job titlevivo:orcidIdORCID idPositionvivo:positionInOrganizationposition in organizationvivo:preferredTitlepreferred titleResearcher Rolevivo:researcherRoleOfresearcher role ofRolevivo:roleContributesTocontributes tovivo:sponsorAwardIdsponsor award idrdf:typetyperdfs:labellabelAgentfoaf:firstNamefirst namefoaf:lastNamelast nameOrganizationPerson2Associate ProfessorPediatricsResearchChildren's Mercy Kansas CityPhDJayVivianJay Vivian0.000000000000000.00000000000000206Vivian, Jay0000-0001-5375-5974Director, Rare Disease Model Research ProgramPIMajor Goals of this study are to develop a cell-based platform using engineered inidcued pluripotent stem cells for assessment of autophagy for use in a pilot-level screen of FDA approved and bioactive small molecule compound library for potential therapies for TBCK Syndrometrue1Director, Rare Disease Model Research ProgramDirector, Rare Disease Model Research Program