Bringing p53 Back: A Prion-Powered Attack on Retinoblastoma.

ABSTRACT: Retinoblastoma (RB) represents the most common primary intraocular malignancy in children, driving a critical need for innovative, targeted therapies that enhance tumor control while preserving vision. Current chemotherapy regimens, such as melphalan, can result in significant systemic toxicity and ocular side effects, underscoring the urgency for safer, more selective treatments. Here, we comprehensively report the design and evaluation of a prion-like self-assembling peptide prodrug (Pri-MP) that exploits the elevated macropinocytic uptake in RB cells to deliver an HDMX-targeting peptide, thereby restoring p53 function. Using single-cell RNA sequencing, we identified a key role for Rac1-PAK1 signaling in driving RB-specific macropinocytosis, which facilitated selective intracellular accu

SUBMITTER: Ma Y 

PROVIDER: S-EPMC12510174 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature