RNAscope in situ hybridization-based method for detecting DUX4 RNA expression in vitro

por Amini Chermahini, Gholamhossein, Rashnonejad, Afrooz, Harper, Scott Q.
Publicado 2019

Construction of permanently inducible miRNA-based expression vectors using site-specific recombinases

por Garwick-Coppens, Sara E, Herman, Adam, Harper, Scott Q
Publicado 2011

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

por Wallace, Lindsay M, Moreo, Andrew, Clark, K Reed, Harper, Scott Q
Publicado 2013

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

por Ansseau, Eugénie, Vanderplanck, Céline, Wauters, Armelle, Harper, Scott Q., Coppée, Frédérique, Belayew, Alexandra
Publicado 2017

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

por Rashnonejad, Afrooz, Amini-Chermahini, Gholamhossein, Taylor, Noah K., Wein, Nicolas, Harper, Scott Q.
Publicado 2020

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

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

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

por Ansseau, Eugénie, Domire, Jacqueline S., Wallace, Lindsay M., Eidahl, Jocelyn O., Guckes, Susan M., Giesige, Carlee R., Pyne, Nettie K., Belayew, Alexandra, Harper, Scott Q.
Publicado 2015

Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy

por Saad, Nizar Y., Al-Kharsan, Mustafa, Garwick-Coppens, Sara E., Chermahini, Gholamhossein Amini, Harper, Madison A., Palo, Andrew, Boudreau, Ryan L., Harper, Scott Q.
Publicado 2021

Meeting report: the 2021 FSHD International Research Congress

por Jagannathan, Sujatha, de Greef, Jessica C., Hayward, Lawrence J., Yokomori, Kyoko, Gabellini, Davide, Mul, Karlien, Sacconi, Sabrina, Arjomand, Jamshid, Kinoshita, June, Harper, Scott Q.
Publicado 2022

Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4

por Eidahl, Jocelyn O., Giesige, Carlee R., Domire, Jacqueline S., Wallace, Lindsay M., Fowler, Allison M., Guckes, Susan M., Garwick-Coppens, Sara E., Labhart, Paul, Harper, Scott Q.
Publicado 2016

Meeting report: the 2020 FSHD International Research Congress

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

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

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

RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model

por Aimiuwu, Osasumwen V., Fowler, Allison M., Sah, Megha, Teoh, Jia Jie, Kanber, Ayla, Pyne, Nettie K., Petri, Sabrina, Rosenthal-Weiss, Chana, Yang, Mu, Harper, Scott Q., Frankel, Wayne N.
Publicado 2020

Is Upregulation of Sarcolipin Beneficial or Detrimental to Muscle Function?

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

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

por Wallace, Lindsay M., Saad, Nizar Y., Pyne, Nettie K., Fowler, Allison M., Eidahl, Jocelyn O., Domire, Jacqueline S., Griffin, Danielle A., Herman, Adam C., Sahenk, Zarife, Rodino-Klapac, Louise R., Harper, Scott Q.
Publicado 2017

A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice

por Stavrou, Marina, Kagiava, Alexia, Choudury, Sarah G., Jennings, Matthew J., Wallace, Lindsay M., Fowler, Allison M., Heslegrave, Amanda, Richter, Jan, Tryfonos, Christina, Christodoulou, Christina, Zetterberg, Henrik, Horvath, Rita, Harper, Scott Q., Kleopa, Kleopas A.
Publicado 2022

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

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

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

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

Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis

por Coulis, Gerald, Jaime, Diego, Guerrero-Juarez, Christian, Kastenschmidt, Jenna M., Farahat, Philip K., Nguyen, Quy, Pervolarakis, Nicholas, McLinden, Katherine, Thurlow, Lauren, Movahedi, Saba, Duarte, Jorge, Sorn, Andrew, Montoya, Elizabeth, Mozaffar, Izza, Dragan, Morgan, Othy, Shivashankar, Joshi, Trupti, Hans, Chetan P., Kimonis, Virginia, MacLean, Adam L., Nie, Qing, Wallace, Lindsay M., Harper, Scott Q., Mozaffar, Tahseen, Hogarth, Marshall W., Bhattacharya, Surajit, Jaiswal, Jyoti K., Golann, David R., Su, Qi, Kessenbrock, Kai, Stec, Michael, Spencer, Melissa J., Zamudio, Jesse R., Villalta, S. Armando
Publicado 2023

Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis

por Coulis, Gerald, Jaime, Diego, Guerrero-Juarez, Christian, Kastenschmidt, Jenna M., Farahat, Philip K., Nguyen, Quy, Pervolarakis, Nicholas, McLinden, Katherine, Thurlow, Lauren, Movahedi, Saba, Hughes, Brandon S., Duarte, Jorge, Sorn, Andrew, Montoya, Elizabeth, Mozaffar, Izza, Dragan, Morgan, Othy, Shivashankar, Joshi, Trupti, Hans, Chetan P., Kimonis, Virginia, MacLean, Adam L., Nie, Qing, Wallace, Lindsay M., Harper, Scott Q., Mozaffar, Tahseen, Hogarth, Marshall W., Bhattacharya, Surajit, Jaiswal, Jyoti K., Golann, David R., Su, Qi, Kessenbrock, Kai, Stec, Michael, Spencer, Melissa J., Zamudio, Jesse R., Villalta, S. Armando
Publicado 2023