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R. Beroukhim

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#gene editing Oct 2026

Abstract C131: Influences of ancestry-associated non-pathogenic germline variants as cancer vulnerabilities

The advent of precision oncology has transformed cancer care. Yet only approximately 25% of patients are eligible for targeted therapies, as current approaches exploit tumors harboring well-characterized somatic driver alterations. This limitation has restricted the broader clinical impact of precision medicine and c...

Nicole Peiris, Sean A. Misek, Angela Chen et al. · 0 citations
Open access Sep 2026

Neoadjuvant PD1 plus CTLA-4 immune checkpoint blockade in surgically resectable recurrent glioblastoma: a randomized surgical window-of-opportunity trial

We conducted a randomized surgical window-of-opportunity trial (NCT04606316) in recurrent, resectable glioblastoma. Between 2021 and 2024, 71 patients were screened, and 63 were randomized (intention-to-treat [ITT] population), and 58 received study treatment. Patients received pre-surgical immune checkpoint blockade (...

Lu Sun, Lauren Markus, Julio C. Sanchez et al. · 0 citations
Aug 2025

O-mannosylation and protein maturation check-points represent therapeutic opportunities in BRAF fusion protein oncogenesis

Fusions between protein-coding genes are common oncogenic drivers across cancers, typically pairing a proto-oncogene with partner that does not independently drive cancer. In all therapeutically actionable fusions, the proto-oncogene is the drug target, the contributions to oncogenicity of the fusion partner have large...

Anna Borgenvik, Sean A. Misek, Daniel Christen et al. · 1 citation
Open access Aug 2026

O-mannosylation and furin processing are critical maturation events for the KIAA1549 protein implicated in pediatric pilocytic astrocytomas

These intrinsic posttranslational modifications of the KIAA1549 ectodomain are critical determinants of the cellular trafficking that lead to activation of the BRAF oncogene, potentially involving a cascade of cleavage events in the Golgi that ultimately release a truncated but fully active BRAF kinase domain into the...

Gloria Kyrila, Serpil Ahmed, Anna Borgenvik et al. · 0 citations
Open access Aug 2026

A compendium of next-generation patient-derived models for diverse cancers

A large-scale international programme—the Human Cancer Models Initiative—is presented, which involved the generation of a resource of 665 next-generation models from 2,780 donors with 25 cancer types and integrated tumour–model whole genome, exome, methylome and transcriptome analyses.

Dina Elharouni, Mushriq Al-Jazrawe, Seongmin Choi et al. · 7 citations
Open access Aug 2026

Splicing genes as main targets of differential splicing.

This work has attempted to identify which genes are most frequently targeted by differential splicing using both downloaded and original RNA-Seq data from several species and tissue types.

T. Tengs, T. LaFramboise, Yossef Baidi et al. · 0 citations

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