Dose-dependent Toxicity of Humanized Renilla reniformis GFP (hrGFP) Limits Its Utility as a Reporter Gene in Mouse Muscle

by Lindsay M Wallace, Andrew Moreo, K Reed Clark, Scott Q Harper
Published 2013-01-01

Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD)

by Eugénie Ansseau, Céline Vanderplanck, Armelle Wauters, Scott Q. Harper, Frédérique Coppée, Alexandra Belayew
Published 2017-03-01

Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

by Afrooz Rashnonejad, Gholamhossein Amini-Chermahini, Noah K. Taylor, Nicolas Wein, Scott Q. Harper
Published 2021-03-01

Conditional over-expression of PITX1 causes skeletal muscle dystrophy in mice

by Sachchida N. Pandey, Jennifer Cabotage, Rongye Shi, Manjusha Dixit, Margret Sutherland, Jian Liu, Stephanie Muger, Scott Q. Harper, Kanneboyina Nagaraju, Yi-Wen Chen
Published 2012-05-01

Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences.

by Eugénie Ansseau, Jacqueline S Domire, Lindsay M Wallace, Jocelyn O Eidahl, Susan M Guckes, Carlee R Giesige, Nettie K Pyne, Alexandra Belayew, Scott Q Harper
Published 2015-01-01

Meeting report: the 2020 FSHD International Research Congress

by Michael Kyba, Robert J. Bloch, Julie Dumonceaux, Scott Q. Harper, Silvère M. van der Maarel, Francis M. Sverdrup, Kathryn R. Wagner, Baziel van Engelen, Yi-Wen Chen
Published 2020-12-01

RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice

by Jian Liu, Lindsay M Wallace, Sara E Garwick-Coppens, Darcée D Sloboda, Carol S Davis, Chady H Hakim, Michael A Hauser, Susan V Brooks, Jerry R Mendell, Scott Q Harper
Published 2014-01-01

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

by Lindsay M. Wallace, Nizar Y. Saad, Nettie K. Pyne, Allison M. Fowler, Jocelyn O. Eidahl, Jacqueline S. Domire, Danielle A. Griffin, Adam C. Herman, Zarife Sahenk, Louise R. Rodino-Klapac, Scott Q. Harper
Published 2018-03-01

Homologous Transcription Factors DUX4 and DUX4c Associate with Cytoplasmic Proteins during Muscle Differentiation.

by Eugénie Ansseau, Jocelyn O Eidahl, Céline Lancelot, Alexandra Tassin, Christel Matteotti, Cassandre Yip, Jian Liu, Baptiste Leroy, Céline Hubeau, Cécile Gerbaux, Samuel Cloet, Armelle Wauters, Sabrina Zorbo, Pierre Meyer, Isabelle Pirson, Dalila Laoudj-Chenivesse, Ruddy Wattiez, Scott Q Harper, Alexandra Belayew, Frédérique Coppée
Published 2016-01-01

A stromal progenitor and ILC2 niche promotes muscle eosinophilia and fibrosis-associated gene expression

by Jenna M. Kastenschmidt, Gerald Coulis, Philip K. Farahat, Phillip Pham, Rodolfo Rios, Therese T. Cristal, Ali H. Mannaa, Rachel E. Ayer, Rayan Yahia, Archis A. Deshpande, Brandon S. Hughes, Adam K. Savage, Carlee R. Giesige, Scott Q. Harper, Richard M. Locksley, Tahseen Mozaffar, S. Armando Villalta
Published 2021-04-01

Is Upregulation of Sarcolipin Beneficial or Detrimental to Muscle Function?

by Naresh C. Bal, Subash C. Gupta, Subash C. Gupta, Meghna Pant, Danesh H. Sopariwala, Geoffrey Gonzalez-Escobedo, Joanne Turner, Joanne Turner, John S. Gunn, John S. Gunn, Christopher R. Pierson, Christopher R. Pierson, Christopher R. Pierson, Scott Q. Harper, Jill A. Rafael-Fortney, Muthu Periasamy, Muthu Periasamy
Published 2021-03-01