Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor

by Juergen Scharner, Sabrina Qi, Frank Rigo, C. Frank Bennett, Adrian R. Krainer
Published 2019-06-01

Manipulation of PK-M mutually exclusive alternative splicing by antisense oligonucleotides

by Zhenxun Wang, Hyun Yong Jeon, Frank Rigo, C. Frank Bennett, Adrian R. Krainer
Published 2012-01-01

Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.

by Yimin Hua, Timothy A Vickers, Brenda F Baker, C Frank Bennett, Adrian R Krainer
Published 2007-04-01

Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1

by Ningyan Hu, Layal Antoury, Timothy M. Baran, Soumya Mitra, C. Frank Bennett, Frank Rigo, Thomas H. Foster, Thurman M. Wheeler
Published 2018-12-01

Evaluation of potential effects of Plastin 3 overexpression and low-dose SMN-antisense oligonucleotides on putative biomarkers in spinal muscular atrophy mice.

by Eike A Strathmann, Miriam Peters, Seyyedmohsen Hosseinibarkooie, Frank W Rigo, C Frank Bennett, Phillip G Zaworski, Karen S Chen, Michael Nothnagel, Brunhilde Wirth
Published 2018-01-01

Mammary Tumor-Associated RNAs Impact Tumor Cell Proliferation, Invasion, and Migration

by Sarah D. Diermeier, Kung-Chi Chang, Susan M. Freier, Junyan Song, Osama El Demerdash, Alexander Krasnitz, Frank Rigo, C. Frank Bennett, David L. Spector
Published 2016-09-01

Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy

by Andrew P. Lieberman, Zhigang Yu, Sue Murray, Raechel Peralta, Audrey Low, Shuling Guo, Xing Xian Yu, Constanza J. Cortes, C. Frank Bennett, Brett P. Monia, Albert R. La Spada, Gene Hung
Published 2014-05-01

Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice

by Dominic Jauvin, Jessina Chrétien, Sanjay K. Pandey, Laurie Martineau, Lucille Revillod, Guillaume Bassez, Aline Lachon, A. Robert McLeod, Geneviève Gourdon, Thurman M. Wheeler, Charles A. Thornton, C. Frank Bennett, Jack Puymirat
Published 2017-06-01

MaTAR25 lncRNA regulates the Tensin1 gene to impact breast cancer progression

by Kung-Chi Chang, Sarah D. Diermeier, Allen T. Yu, Lily D. Brine, Suzanne Russo, Sonam Bhatia, Habeeb Alsudani, Karen Kostroff, Tawfiqul Bhuiya, Edi Brogi, Darryl J. Pappin, C. Frank Bennett, Frank Rigo, David L. Spector
Published 2020-12-01

Effects on murine behavior and lifespan of selectively decreasing expression of mutant huntingtin allele by supt4h knockdown.

by Hui-Min Cheng, Yijuang Chern, I-Hui Chen, Chia-Rung Liu, Sih-Huei Li, Seung J Chun, Frank Rigo, C Frank Bennett, Ning Deng, Yanan Feng, Chyuan-Sheng Lin, Yu-Ting Yan, Stanley N Cohen, Tzu-Hao Cheng
Published 2015-03-01

Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.HttQ111/+ mouse model of Huntington's disease.

by Sydney R Coffey, Robert M Bragg, Shawn Minnig, Seth A Ament, Jeffrey P Cantle, Anne Glickenhaus, Daniel Shelnut, José M Carrillo, Dominic D Shuttleworth, Julie-Anne Rodier, Kimihiro Noguchi, C Frank Bennett, Nathan D Price, Holly B Kordasiewicz, Jeffrey B Carroll
Published 2017-01-01

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.

by Niels H Skotte, Amber L Southwell, Michael E Østergaard, Jeffrey B Carroll, Simon C Warby, Crystal N Doty, Eugenia Petoukhov, Kuljeet Vaid, Holly Kordasiewicz, Andrew T Watt, Susan M Freier, Gene Hung, Punit P Seth, C Frank Bennett, Eric E Swayze, Michael R Hayden
Published 2014-01-01

α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease

by Tracy A. Cole, Hien Zhao, Timothy J. Collier, Ivette Sandoval, Caryl E. Sortwell, Kathy Steece-Collier, Brian F. Daley, Alix Booms, Jack Lipton, Mackenzie Welch, Melissa Berman, Luke Jandreski, Danielle Graham, Andreas Weihofen, Stephanie Celano, Emily Schulz, Allyson Cole-Strauss, Esteban Luna, Duc Quach, Apoorva Mohan, C. Frank Bennett, Eric E. Swayze, Holly B. Kordasiewicz, Kelvin C. Luk, Katrina L. Paumier
Published 2021-03-01