2014
Gene Expression and Mutation Analysis (GEMA) –Guided Precision Medicine Targeting PARP1 to Induce Synthetic Lethality in DNA-PK –Deficient Quiescent and BRCA-Deficient Proliferating Leukemia Stem and Progenitor Cells
Nieborowska-Skorska M, Sullivan K, Podszywalow-Bartnicka P, Hoser G, Bolton-Gillespie E, Cramer-Morales K, Slupianek A, Zhou C, Cerny-Reiterer S, Stoklosa T, Sykes S, Valent P, Civin C, Muschen M, Minden M, Eppert K, Skorski T. Gene Expression and Mutation Analysis (GEMA) –Guided Precision Medicine Targeting PARP1 to Induce Synthetic Lethality in DNA-PK –Deficient Quiescent and BRCA-Deficient Proliferating Leukemia Stem and Progenitor Cells. Blood 2014, 124: 480. DOI: 10.1182/blood.v124.21.480.480.Peer-Reviewed Original ResearchQuiescent leukemia stem cellsLeukemia progenitor cellsLeukemia stem cellsIndividual patientsPARP1 inhibitorsBCR-ABL1MLL-AF9Progenitor cellsHomologous recombination repairSurvival of miceAML1-ETOAnti-tumor treatmentBone marrow cellsQuiescent cellsMutation analysisCML-CPDisease burdenImmunodeficient miceTumor initiatingLeukemia xenograftsReduced colony formationPatientsPharmacological inhibitionDNA double-strand breaksRepair mechanisms
2007
Preclinical Evaluation of CBP/β-catenin Inhibition as a New Strategy for Drug Resistant Acute Lymphoblastic Leukemia.
Kim Y, Park E, Lorentzen C, De La Torre B, Hsieh Y, Whang H, Klemm L, Nguyen C, McMillan M, Teo J, Muschen M, Kahn M. Preclinical Evaluation of CBP/β-catenin Inhibition as a New Strategy for Drug Resistant Acute Lymphoblastic Leukemia. Blood 2007, 110: 1596. DOI: 10.1182/blood.v110.11.1596.1596.Peer-Reviewed Original ResearchAcute lymphoblastic leukemiaMedian survival timeStandard chemotherapyICG-001Expression of survivinLymphoblastic leukemiaLeukemia cellsXenograft modelDrug-resistant acute lymphoblastic leukemiaSmall molecule inhibitor ICG-001NOD/SCID xenograft modelHuman preCBP/β-cateninPromising therapeutic principleBlood count analysisSCID xenograft modelSurvival of miceNovel therapeutic optionsNew treatment modalitiesΒ-cateninDrug-resistant leukemia cellsΒ-catenin inhibitionHigh death rateResistant leukemia cellsSurvivin mRNA expression