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Developing Schwann Specific Promoters
TS-005616 — This new invention is a Schwann cell-specific promoter that allows detailed, selective gene expression in Schwann cells while maintaining compatibility with AAV vectors, a limitation found in the current promoter systems for Schwann cells. Not only will this address a major field-wide barrier, but is also applicable to gene therapies for demyelinating neuropathies, including CMT1A, CMT4, CMTX, NF1, NF2, and inflammatory or injury-associated Schwann-cell disorders. Additionally, this invention will allow an optimized AAV9-KDAR expression cassette for CMT1B, incorporating the lead SSP. This finalized therapeutic construct will be ready for in vivo evaluation in our CMT1B mouse models, enabling selection of the lead candidate for subsequent IND-enabling studies and progression toward clinical translation.
  • College:
  • Inventors: Rashnonejad, Afrooz; Zhu, Jingting
  • Licensing Officer: Eidahl, Jocelyn

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