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Engineering a human-based translational activator for targeted protein expression restoration.

Riley W Sinnott1, Ani Solanki2, Anitha P Govind3

  • 1Department of Chemistry, The University of Chicago, 5735 S. Ellis Ave., Chicago, IL 60637.

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This study introduces a new method to boost protein production for diseases like Dravet syndrome. A novel translational activator improved survival and reduced seizures in a mouse model by increasing SCN1a protein levels.

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Area of Science:

  • Genetics and Molecular Biology
  • Neuroscience
  • Therapeutic Development

Background:

  • Haploinsufficiency, caused by deficient gene expression, requires new therapeutic strategies.
  • Increasing protein translation from existing mRNA offers a promising approach to restore physiological levels.
  • Previous methods have not been tested in vivo for neurological disorders.

Purpose of the Study:

  • To investigate the efficacy of a translational activator in a mouse model of Dravet syndrome.
  • To engineer human proteins for programmable, guide RNA-directed mRNA translation using the CRISPR-Cas Inspired RNA-targeting System (CIRTS).
  • To assess if increased translation of SCN1a mRNA can ameliorate disease phenotype.

Main Methods:

  • Development of the CRISPR-Cas Inspired RNA-targeting System (CIRTS) platform with engineered human proteins.
  • Identification of a compact translational activator (CIRTS-4GT3) capable of enhancing endogenous transcript translation.
  • Adeno-associated virus (AAV)-mediated delivery of CIRTS-4GT3 targeting SCN1a mRNA in a Dravet syndrome mouse model.

Main Results:

  • CIRTS-4GT3 demonstrated targeted, sustained translation increases up to 100% for epilepsy and neurodevelopmental disorder-relevant transcripts.
  • AAV-delivery of CIRTS-4GT3 in the Dravet syndrome model successfully increased SCN1a translation.
  • Significant improvements in survivability and seizure threshold were observed in treated mice.

Conclusions:

  • This work validates translational activation as a viable strategy for addressing SCN1a haploinsufficiency.
  • The CIRTS platform enables programmable enhancement of protein production for therapeutic benefit.
  • Translational activation holds significant preclinical potential for treating neurological haploinsufficiency disorders.