June 2013
Volume 54, Issue 15
Free
ARVO Annual Meeting Abstract  |   June 2013
Investigation of Gene Therapy Using Immortalized Cells Derived from a Gelatinous Drop-Like Corneal Dystrophy Patient
Author Affiliations & Notes
  • Koji Kitazawa
    Kyoto Prefectural Univ of Med, Kyoto, Japan
  • Satoshi Kawasaki
    Kyoto Prefectural Univ of Med, Kyoto, Japan
  • Keita Aoi
    Kyoto Prefectural Univ of Med, Kyoto, Japan
    Doshisya University, Kyoto, Japan
  • Katsuhiko Shinomiya
    Kyoto Prefectural Univ of Med, Kyoto, Japan
  • Akira Matsuda
    Juntendo University, Tokyo, Japan
  • Toshinari Funaki
    Juntendo University, Tokyo, Japan
  • Mina Nakatsukasa
    Kyoto Prefectural Univ of Med, Kyoto, Japan
  • Junji Hamuro
    Kyoto Prefectural Univ of Med, Kyoto, Japan
  • Akira Murakami
    Juntendo University, Tokyo, Japan
  • Shigeru Kinoshita
    Kyoto Prefectural Univ of Med, Kyoto, Japan
  • Footnotes
    Commercial Relationships Koji Kitazawa, None; Satoshi Kawasaki, None; Keita Aoi, None; Katsuhiko Shinomiya, None; Akira Matsuda, None; Toshinari Funaki, None; Mina Nakatsukasa, None; Junji Hamuro, None; Akira Murakami, SEED(Japan) JP4855782 (P), SEED(Japan) JP5132958 (P); Shigeru Kinoshita, Senju Pharmaceutical Co (P), Santen Pharmaceutical Co (P), Otsuka Pharmaceutical Co (C), Alcon (R), AMO (R), HOYA (R)
  • Footnotes
    Support None
Investigative Ophthalmology & Visual Science June 2013, Vol.54, 4559. doi:https://doi.org/
  • Views
  • Share
  • Tools
    • Alerts
      ×
      This feature is available to authenticated users only.
      Sign In or Create an Account ×
    • Get Citation

      Koji Kitazawa, Satoshi Kawasaki, Keita Aoi, Katsuhiko Shinomiya, Akira Matsuda, Toshinari Funaki, Mina Nakatsukasa, Junji Hamuro, Akira Murakami, Shigeru Kinoshita; Investigation of Gene Therapy Using Immortalized Cells Derived from a Gelatinous Drop-Like Corneal Dystrophy Patient. Invest. Ophthalmol. Vis. Sci. 2013;54(15):4559. doi: https://doi.org/.

      Download citation file:


      © ARVO (1962-2015); The Authors (2016-present)

      ×
  • Supplements
Abstract

Purpose: Gelatinous drop-like corneal dystrophy (GDLD) is an autosomal recessive inheritance disease caused by biallelic loss-of-function mutations within the tumor-associated calcium signal transducer 2 (TACSTD2) gene. We previously established two immortalized cell lines via the lentiviral introduction of the SV40 large T antigen and hTERT genes into the corneal and conjunctival epithelial cells of GDLD patients. The purpose of this present study was to assess whether or not a gene therapy using these two cells lines is effective for the treatment of GDLD patients.

Methods: The lentivirus vector harboring the wild-type TACSTD2 gene was produced and transduced to the two immortalized cell lines described above. Epithelial barrier function of the two cell lines was investigated by means of trans-epithelial resistance (TER) analysis and dye permeabilization testing using fluorescein in order to assess whether or not the transduction of the wild-type TACSTD2 gene actually normalizes the disease situation of GDLD cornea.

Results: The transduction efficiency of the wild-type TACSTD2 gene to the immortalized cell lines was approximately 80%. TER of the immortalized cell lines was increased after the transduction of the wild-type TACSTD2 gene. The permeability of fluorescein in the immortalized cell lines was decreased after the transduction of the wild-type TACSTD2 gene.

Conclusions: The findings of this study show that transduction of the wild-type TACSTD2 gene normalizes the disease situation of GDLD corneas to some extent, thus indicating that gene therapy may prove to be a promising treatment for GDLD.

Keywords: 482 cornea: epithelium • 494 degenerations/dystrophies • 538 gene transfer/gene therapy  
×
×

This PDF is available to Subscribers Only

Sign in or purchase a subscription to access this content. ×

You must be signed into an individual account to use this feature.

×