Treatment of a Mouse Model of ALS by In Vivo Base Editing

Colin K.W. Lim, Michael Gapinske, Alexandra K. Brooks, Wendy S. Woods, Jackson E. Powell, M. Alejandra Zeballos C., Jackson Winter, Pablo Perez-Pinera, Thomas Gaj

Research output: Contribution to journalArticle


Lim et al. establish a trans-splicing system to deliver CRISPR base editors in vivo that enables treatment of a mouse model of ALS. They show that base editing can increase survival and slow the progression of disease.

Original languageEnglish (US)
JournalMolecular Therapy
StateAccepted/In press - Jan 1 2020



  • AAV
  • ALS
  • base editor
  • CRISPR-Cas9
  • gene editing
  • gene therapy
  • neurodegeneration
  • SOD1
  • split intein

ASJC Scopus subject areas

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

Cite this

Lim, C. K. W., Gapinske, M., Brooks, A. K., Woods, W. S., Powell, J. E., Zeballos C., M. A., Winter, J., Perez-Pinera, P., & Gaj, T. (Accepted/In press). Treatment of a Mouse Model of ALS by In Vivo Base Editing. Molecular Therapy.