Investigating the role of ZBTB7A in AML differentiation

The proposed animal studies are essential to evaluate the in vivo effects of AML engraftment, disease progression, and therapeutic response, where systemic treatment exposure and leukemia interactions with the hematopoietic microenvironment cannot be adequately modeled in vitro. This includes the ability to assess the in vivo delivery efficiency and therapeutic potential of CpG-conjugated ASOs targeting of the long ZBTB7A 3′UTR isoform in AML. In addition, the NSG-SGM3 mice are particularly well suited for these studies because they are the most robust model to support engraftment of primary AML patient-derived xenografts, enabling faithful assessment of leukemia growth, treatment response, and survival while preserving the biologic heterogeneity of patient samples. These models are therefore required to determine the effects of ELAVL1/ELAVL4 perturbation in Aim 1 and CpG-conjugated ASO targeting of the long ZBTB7A 3′UTR isoform on leukemia burden and survival in Aim 2. Project Number: 1R01CA295672-01A1 | Fiscal Year: 2026 | NIH Institute/Center: National Cancer Institute (NCI) | Principal Investigator: Eric Wang | Institution: JACKSON LABORATORY, BAR HARBOR, ME | Award Amount: $543,984 | Activity Code: R01 | Study Section: Gene Regulation in Cancer Study Section[GRIC] View on NIH RePORTER: https://reporter.nih.gov/project-details/11296247