Edited by Lora Hooper, The University of Texas Southwestern Medical Center, Dallas, TX; received March 18, 2025; accepted August 4, 2025 ...
Following the administration of BB-301, Cohort 1 patients experienced significant continuing reductions in dysphagic symptom burden, post-swallow residue accumulation, time required to consume fixed ...
The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace ... The proprietary “Silence and ...
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