Research ArticleRett Syndrome
Pharmacological enhancement of KCC2 gene expression exerts therapeutic effects on human Rett syndrome neurons and Mecp2 mutant mice
- View ORCID ProfileXin Tang1,
- Jesse Drotar1,
- View ORCID ProfileKeji Li2,
- View ORCID ProfileCullen D. Clairmont1,
- Anna Sophie Brumm3,
- View ORCID ProfileAustin J. Sullins2,
- Hao Wu4,
- Xiaoxiao Shawn Liu1,
- Jinhua Wang5,
- Nathanael S. Gray5,
- View ORCID ProfileMriganka Sur2 and
- View ORCID ProfileRudolf Jaenisch1,6,*
- 1Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
- 2Picower Institute for Learning and Memory, Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
- 3Francis Crick Institute, London NW1 1AT, UK .
- 4Fulcrum Therapeutics, Cambridge, MA 02139, USA.
- 5Department of Cancer Biology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA.
- 6Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02142, USA.
- ↵*Corresponding author. Email: jaenisch{at}wi.mit.edu
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Science Translational Medicine 31 Jul 2019:
Vol. 11, Issue 503, eaau0164
DOI: 10.1126/scitranslmed.aau0164
Vol. 11, Issue 503, eaau0164
DOI: 10.1126/scitranslmed.aau0164
Xin Tang
1Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
Jesse Drotar
1Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
Keji Li
2Picower Institute for Learning and Memory, Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Cullen D. Clairmont
1Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
Anna Sophie Brumm
3Francis Crick Institute, London NW1 1AT, UK .
Austin J. Sullins
2Picower Institute for Learning and Memory, Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Hao Wu
4Fulcrum Therapeutics, Cambridge, MA 02139, USA.
Xiaoxiao Shawn Liu
1Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
Jinhua Wang
5Department of Cancer Biology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA.
Nathanael S. Gray
5Department of Cancer Biology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA.
Mriganka Sur
2Picower Institute for Learning and Memory, Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Rudolf Jaenisch
1Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
6Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02142, USA.
