T. Haferlach

Unbiased, systematic surfaceome profiling of Acute Leukemia to identify novel immunotherapy targets

Cell surface proteomics provides a direct topological assessment of the outer membrane of cells and enables the capture of low abundance proteins that may be missed by whole cell proteomics. Here we present an unbiased atlas of the whole cell and surface proteomes of 25 commonly used leukemic cell lines, encompassing b...

MDS without clonal marker: when depth outperforms breadth

Csnk1a1 E98 mutations rewire signaling and metabolism in del(5q) myelodysplastic neoplasms.

Deletion of chromosome 5q [del(5q)] is the most common cytogenetic abnormality in myelodysplastic neoplasms (MDS) and results in haploinsufficiency of multiple genes, including CSNK1A1. Recurrent CSNK1A1 mutations, predominantly affecting the E98 hotspot, occur almost exclusively in del(5q) MDS and are associated with...

Dual MYC and GSPT1 Protein Degrader for MYC-Driven Hematologic Malignancies.

The data support the development of the MYC/GSPT1 degrader GT19630 as a therapeutic strategy of MYC-driven hematologic malignancies as a therapeutic strategy of therapy-resistant hematologic malignancies.

MAP3K14 (NIK) synonymous variants drive altered splicing to activate NF-κB in mature B-cell malignancies.

The spectrum of genetic lesions that result in activation of NF‑κB in mature B‑cell malignancies is expanded and highlights the incomplete understanding of NF‑κB drivers in mature B‑cell malignancies.

The novel hypomethylating agent NTX-301 reprograms epigenetic and Hippo signaling pathways and exhibits pre-clinical activity in venetoclax-resistant and TP53-mutant AML.

Data suggest that NTX-301 exerts potent anti-leukemia activities superior to 5-AZA and synergizes with VEN in VEN-resistant and TP53-mutant AML, in part by suppressing DNMT1 and inducing DNA damage responses and apoptosis, by inducing p53 signaling and demethylating LATS1/2, thus activating Hippo signaling.