A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion

Yu Chen Chen, Ning Xin Ma, Zi Fei Pei, Zheng Wu, Fabricio H. Do-Monte, Susan Keefe, Emma Yellin, Miranda S. Chen, Jiu Chao Yin, Grace Lee, Angélica Minier-Toribio, Yi Hu, Yu Ting Bai, Kathryn Lee, Gregory J. Quirk, Gong Chen

Research output: Contribution to journalArticle

6 Scopus citations

Abstract

After ischemic brain injury, many neurons die but surviving astrocytes become activated and proliferative. Using NeuroD1 AAV-based gene therapy, Chen and colleagues demonstrate robust neuroregeneration through direct astrocyte-to-neuron conversion and significantly improved functional recovery. This study provides a new paradigm for brain repair using in vivo cell conversion technology.

Original languageEnglish (US)
JournalMolecular Therapy
DOIs
StateAccepted/In press - Jan 1 2019

All Science Journal Classification (ASJC) codes

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery

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    Chen, Y. C., Ma, N. X., Pei, Z. F., Wu, Z., Do-Monte, F. H., Keefe, S., Yellin, E., Chen, M. S., Yin, J. C., Lee, G., Minier-Toribio, A., Hu, Y., Bai, Y. T., Lee, K., Quirk, G. J., & Chen, G. (Accepted/In press). A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2019.09.003