grant

Gene Therapy for ALPK3 Cardiomyopathy Using MiniALPK3

Organization UNIVERSITY OF CALIFORNIA, SAN DIEGOLocation LA JOLLA, UNITED STATESPosted 15 Jul 2024Deadline 31 May 2028
NIHUS FederalResearch GrantFY202521+ years oldAAV vectorAAV-based vectorATP-protein phosphotransferaseAddressAdeno-Associated VirusesAdultAdult HumanAge MonthsAllelesAllelic LossAllelomorphsAmino AcidsAnimal ModelAnimal Models and Related StudiesAsymmetric Septal HypertrophyBase PairingBirthCardiacCardiac AbnormalitiesCardiac Muscle CellsCardiac MyocytesCardiocyteCardiomyopathiesCardiovascular DiseasesCodeCoding SystemCompensationComplementary DNACongestive CardiomyopathyDNA TherapyDNA mutationDataData BasesDatabasesDependoparvovirusDependovirusDevelopmentDiagnosisDilated CardiomyopathyExhibitsExonsFamilyFibrosis in the heartFibrosis in the myocardiumFibrosis within the heartFibrosis within the myocardiumFibrotic myocardiumGene Transfer ClinicalGenesGenetic ChangeGenetic InterventionGenetic MaterialsGenetic defectGenetic mutationGoalsHeartHeart AbnormalitiesHeart Muscle CellsHeart failureHeart myocyteHereditary ventricular hypertrophyHeterozygoteHumanHypertrophic CardiomyopathyHypertrophic Obstructive CardiomyopathyIdiopathic Hypertrophic Subvalvular StenosisIdiopathic hypertrophic subaortic stenosisIndividualKO miceKinase Family GeneKinasesKnock-out MiceKnockout MiceLengthLoss of HeterozygosityMethodsMiceMice MammalsModern ManMolecularMurineMusMuscleMuscle TissueMutationMyocardial DiseasesMyocardial DisorderMyocardiopathiesMyocardiumNamesNull MouseParturitionPathogenicityPathologicPatientsPhenotypePhosphotransferase GenePhosphotransferasesPlayPremature MortalityProtein KinaseResearchRoleTherapeuticTimeTransphosphorylasesVariantVariationViral PackagingViral VectorVirus Packagingsadeno associated virus groupadeno-associated viral vectoradeno-associated virus vectoradulthoodaminoacidcDNAcardiac failurecardiac fibrosiscardiac functioncardiac musclecardiomyocytecardiovascular disordercohortcoronary fibrosisdata basedesigndesigningdevelopmentalearly childhoodearly onseteffective therapyeffective treatmentfibrotic heartfunction of the heartgene repair therapygene replacementgene replacement therapygene therapygene-based therapygenetic therapygenome mutationgenomic therapyglycogen synthase a kinaseheart fibrosisheart functionheart muscleheterozygosityhuman diseasehydroxyalkyl protein kinasehypertrophic myocardiopathyin vivoinsightloss of functionloss of function mutationmodel of animalmouse modelmurine modelmuscularmyocardial fibrosismyocardium diseasemyocardium disordernamenamednamingnovelphosphorylase b kinase kinasepostnatalprematureprematuritypreventpreventingsocial roletreatment strategy
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Description preview

PROJECT SUMMARY
Alpha Protein Kinase 3 (ALPK3) is an atypical protein kinase belonging to the alpha (α)-kinase family. Loss-of-

function mutations in ALPK3 result in human cardiomyopathy. Currently, no specific treatments are available

for patients diagnosed with ALPK3 cardiomyopathy. To understand the in vivo role of ALPK3, we generated

Alpk3…

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Gene Therapy for ALPK3 Cardiomyopathy Using MiniALPK3 — UNIVERSITY OF CALIFORNIA, SAN DIEGO | UNITED STATES | Jul 2024 | Dev Procure