NF1

[Molecular Therapy-Nucleic Acids] Initial in silico analysis predicted exons that can be skipped with minimal loss of neurofibromin function, which was confirmed with in vitro assessments utilizing an neurofibromatosis type 1 (Nf1) cDNA-based functional screening system.

[Cell Reports] The authors developed an iPSC-based neural crest-Schwann cell in vitro differentiation system and constructed a lineage expression roadmap for the analysis of different 2D and 3D neurofibroma models.

[Nature Communications] The authors described identification of 1089 gene-centric common insertion sites (gCIS) from transposon-based screens in 8 mouse models of breast cancer (BC). Most gCIS showed single allele disruption and many mapped to genomic regions showing high-frequency hemizygous loss in human BC.

[Developmental Cell] Scientists provided the proof-of-concept evidence for preventing pediatric low-grade gliomas before tumor-associated neurological damage entered an irreversible phase.

[Cancer Cell] Researchers compared the efficacies of sequential and/or combinatorial regimens in subcutaneous murine models of melanoma driven by BrafV600, Nras, or Nf1 mutations as well as colorectal and pancreatic carcinoma driven by KrasG12C.

[SpringWorks Therapeutics, Inc.] SpringWorks Therapeutics, Inc. announced that the company will be evaluating mirdametinib, an investigational MEK inhibitor, in a platform study exploring the compound both as a monotherapy and as a combination therapy in advanced solid tumors harboring MAPK-activating mutations.