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1250 articles, sorted by publication time.

Gene Medicine · global

One Dose to Correct a Typo at the RNA Level: New Rett Syndrome Therapy Takes a Preclinical Step Forward

Shape Therapeutics and the Rett Syndrome Research Trust are expanding their collaboration, using AI to select guide RNAs and harnessing cells’ own ADAR enzymes to correct MECP2 mutations. Mouse studies showed RNA editing in the brain and symptom improvement, but the human dose, safety, and eligible patient population remain to be clarified.

Cancer Treatment · global

First Off-the-Shelf TCR-NK Cell Therapy Enters Human Testing, but Patient Experience Cannot Yet Substitute for Efficacy Data

The first patient with an advanced solid tumor has been dosed in the Phase 1 trial of ZI-MA4-1. The therapy seeks to combine precise cancer recognition with the advantages of off-the-shelf manufacturing, but at present only the trial’s initiation can be confirmed; no objective tumor response results are yet available.