DOI: 10.1126/scitranslmed.3007529. Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a <i>C9ORF72</i> Repeat Expansion

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

10.1126/scitranslmed.3007529

Crossref journal-article

American Association for the Advancement of Science (AAAS)

Science Translational Medicine (221)

Abstract

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

Bibliography

Sareen, D., OâRourke, J. G., Meera, P., Muhammad, A. K. M. G., Grant, S., Simpkinson, M., Bell, S., Carmona, S., Ornelas, L., Sahabian, A., Gendron, T., Petrucelli, L., Baughn, M., Ravits, J., Harms, M. B., Rigo, F., Bennett, C. F., Otis, T. S., Svendsen, C. N., & Baloh, R. H. (2013). Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion. Science Translational Medicine, 5(208).

Authors 20

Dhruv Sareen (first)
Jacqueline G. O’Rourke (additional)
Pratap Meera (additional)
A. K. M. G. Muhammad (additional)
Sharday Grant (additional)
Megan Simpkinson (additional)
Shaughn Bell (additional)
Sharon Carmona (additional)
Loren Ornelas (additional)
Anais Sahabian (additional)
Tania Gendron (additional)
Leonard Petrucelli (additional)
Michael Baughn (additional)
John Ravits (additional)
Matthew B. Harms (additional)
Frank Rigo (additional)
C. Frank Bennett (additional)
Thomas S. Otis (additional)
Clive N. Svendsen (additional)
Robert H. Baloh (additional)

References 50 Referenced 598

Dates

Type	When
Created	11 years, 10 months ago (Oct. 23, 2013, 2:25 p.m.)
Deposited	1 year, 7 months ago (Jan. 16, 2024, 1:31 p.m.)
Indexed	7 minutes ago (Aug. 30, 2025, 10:59 p.m.)
Issued	11 years, 10 months ago (Oct. 23, 2013)
Published	11 years, 10 months ago (Oct. 23, 2013)
Published Print	11 years, 10 months ago (Oct. 23, 2013)

Funders 0

None

BibTeX

@article{Sareen_2013, title={Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion}, volume={5}, ISSN={1946-6242}, url={http://dx.doi.org/10.1126/scitranslmed.3007529}, DOI={10.1126/scitranslmed.3007529}, number={208}, journal={Science Translational Medicine}, publisher={American Association for the Advancement of Science (AAAS)}, author={Sareen, Dhruv and O’Rourke, Jacqueline G. and Meera, Pratap and Muhammad, A. K. M. G. and Grant, Sharday and Simpkinson, Megan and Bell, Shaughn and Carmona, Sharon and Ornelas, Loren and Sahabian, Anais and Gendron, Tania and Petrucelli, Leonard and Baughn, Michael and Ravits, John and Harms, Matthew B. and Rigo, Frank and Bennett, C. Frank and Otis, Thomas S. and Svendsen, Clive N. and Baloh, Robert H.}, year={2013}, month=oct }

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