2020
Utility of spontaneous animal models of Alzheimer’s disease in preclinical efficacy studies
Zeiss CJ. Utility of spontaneous animal models of Alzheimer’s disease in preclinical efficacy studies. Cell And Tissue Research 2020, 380: 273-286. PMID: 32337614, DOI: 10.1007/s00441-020-03198-6.Peer-Reviewed Original ResearchConceptsHuman Alzheimer's diseaseSpontaneous animal modelAlzheimer's diseaseAnimal modelsBiomarker progressionProgression of neuropathologyLate-onset Alzheimer's diseasePreclinical efficacy studiesHuman clinical trialsOnset Alzheimer's diseaseUsable outcome measuresAD-associated mutationsFamilial Alzheimer's diseaseNon-human primatesAmyloid neuropathologyInterventional studyClinical trialsSpontaneous modelHuman trialsOutcome measuresTherapeutic successPotential therapyNew therapiesRodent studiesEfficacy studies
2019
An ABCA4 loss-of-function mutation causes a canine form of Stargardt disease
Mäkeläinen S, Gòdia M, Hellsand M, Viluma A, Hahn D, Makdoumi K, Zeiss CJ, Mellersh C, Ricketts SL, Narfström K, Hallböök F, Ekesten B, Andersson G, Bergström TF. An ABCA4 loss-of-function mutation causes a canine form of Stargardt disease. PLOS Genetics 2019, 15: e1007873. PMID: 30889179, PMCID: PMC6424408, DOI: 10.1371/journal.pgen.1007873.Peer-Reviewed Original ResearchMeSH KeywordsAmino Acid SequenceAnimalsATP Binding Cassette Transporter, Subfamily A, Member 4ATP-Binding Cassette TransportersBase SequenceCodon, NonsenseDisease Models, AnimalDog DiseasesDogsFemaleGenes, RecessiveHomozygoteHumansLipofuscinMacular DegenerationMaleMicroscopy, FluorescenceModels, MolecularMutagenesis, InsertionalMutationPedigreeProtein ConformationRetinaStargardt DiseaseWhole Genome SequencingConceptsRetinal pigment epitheliumStargardt diseaseAutosomal recessive retinal degenerative diseaseRetinal degenerationABCA4 geneVisual impairmentCentral visual impairmentFull-length ABCA4 proteinFunction mutationsLabrador Retriever dogsLarge animal modelRetinal degenerative diseasesAutosomal recessive retinal degenerationMember 4 geneRecessive retinal degenerationStandard treatmentClinical trialsClinical signsLipofuscin depositsPigment epitheliumAnimal modelsCanine modelUnaffected dogsAffected dogsCone photoreceptors
2018
Pathology Study Design, Conduct, and Reporting to Achieve Rigor and Reproducibility in Translational Research Using Animal Models.
Everitt JI, Treuting PM, Scudamore C, Sellers R, Turner PV, Ward JM, Zeiss CJ. Pathology Study Design, Conduct, and Reporting to Achieve Rigor and Reproducibility in Translational Research Using Animal Models. ILAR Journal 2018, 59: 4-12. PMID: 30624739, DOI: 10.1093/ilar/ily020.Peer-Reviewed Original ResearchConceptsAnimal modelsAnimal model-based researchAnimal study dataPreclinical animal modelsHuman clinical experienceHuman clinical trialsPreclinical animal studiesAnimal-based studiesPreclinical animal experimentsPreclinical investigatorsClinical trialsPotential therapyAnimal studiesClinical experiencePathology analysisPathology practiceStudy designAnimal experimentsPathology methodsTranslational researchTissue collectionLimited concordanceComparative pathologistsReproducibility of dataToxicologic Pathology
2014
Preclinical Safety Evaluation of a Recombinant AAV8 Vector for X-Linked Retinoschisis After Intravitreal Administration in Rabbits
Marangoni D, Wu Z, Wiley HE, Zeiss CJ, Vijayasarathy C, Zeng Y, Hiriyanna S, Bush RA, Wei LL, Colosi P, Sieving PA. Preclinical Safety Evaluation of a Recombinant AAV8 Vector for X-Linked Retinoschisis After Intravitreal Administration in Rabbits. Human Gene Therapy 2014, 25: 202-211. PMID: 25211193, PMCID: PMC4275775, DOI: 10.1089/humc.2014.067.Peer-Reviewed Original ResearchConceptsIntravitreal administrationDose groupEye groupClinical trialsOcular inflammatory reactionRs1-KO miceHigh-dose groupPreclinical safety evaluationNew Zealand white rabbitsAdeno-associated viral vectorZealand white rabbitsIrreversible tissue damageNovel gene therapy approachVitreous infiltratesIntraocular inflammationOcular inflammationInflammatory infiltrateOphthalmological examinationEye doseInflammatory cellsIntravitreal routeOcular safetyClinical signsCommon causeHistopathological analysis