Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion-Reference-Cited by-同舟云学术

Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion

Published:2013-10-23 Issue:208 Volume:5 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Sareen Dhruv¹²,O’Rourke Jacqueline G.¹,Meera Pratap³,Muhammad A. K. M. G.¹,Grant Sharday¹,Simpkinson Megan¹,Bell Shaughn¹,Carmona Sharon¹,Ornelas Loren¹,Sahabian Anais¹,Gendron Tania⁴,Petrucelli Leonard⁴,Baughn Michael⁵,Ravits John⁵,Harms Matthew B.⁶,Rigo Frank⁷,Bennett C. Frank⁷,Otis Thomas S.³,Svendsen Clive N.¹²,Baloh Robert H.¹⁸

Affiliation:

1. Regenerative Medicine Institute, Cedars-Sinai Medical Center, 8700 Beverly Boulevard, Los Angeles, CA 90048, USA.

2. Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, CA 90048, USA.

3. Department of Neurobiology, University of California, Los Angeles, Los Angeles, CA 90095, USA.

4. Mayo Clinic, Jacksonville, FL 32224, USA.

5. Department of Neurology, University of California, San Diego, 9500 Gilman Drive, La Jolla, CA 92093, USA.

6. Department of Neurology, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.

7. Isis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

8. Department of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA 90048, USA.

Abstract

Antisense oligonucleotides can correct disease-specific phenotypes in cultured motor neurons differentiated from iPSCs derived from ALS patients with a C9ORF72 repeat expansion.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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