Tyrosine Peptides Alleviates Multifaceted Toxicity Linked to Expanded CGG Repeats in Fragile X‑Associated Tremor/Ataxia Syndrome.

ABSTRACT: Fragile X-associated Tremor/Ataxia Syndrome (FXTAS)/Fragile X-associated Primary Ovarian Insufficiency (FXPOI) are conditions caused by an elevated level of CGG repeats in the 5' untranslated region (5'-UTR) of the Fragile X Mental Retardation 1 (FMR1) gene. These trinucleotide extended RNA repeats [r-(CGG)_exp] produce toxic homopolymeric proteins in neuronal cells via repeat-associated non-ATG (RAN) translation or formation of RNA foci. A promising therapeutic approach for such conditions involves identifying potent modulators, particularly those with high binding affinity and selectivity toward these expanded RNA repeats. We investigated the therapeutic potential of homoaromatic tyrosine peptides (2Y, 3Y, and 4Y) toward CGG repeat RNA by employing biophysical and cell model-ba

SUBMITTER: Singh K 

PROVIDER: S-EPMC12171881 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature