Abstracts, Seminars, and Meetings Attended by the Wellstone Group

2015 - 2017

"Using genome-wide CRISPR libraries to identify modifiers of FSHD", 
A. Lek, M. Lek, J. Criscione, A. Pakula, Y. Zhang, O. King, P. Jones, L. Kunkel.
MDA Scientific Conference. Hyatt Regency Crystal City. Arlington VA, March 2017.

"Translating DUX4-targeted RNAi therapy for FSHD", 
L.M. Wallace, D.A. Griffin, N.K. Pyne, J.s. Domirre, L.R. Rodino-Klapac, and S.Q. Harper. 
2017 Muscular Dystrophy Association Scientific Conference, Arlington, VA, March 2017.

"Identification and Characterizatin of DUX4 Post-Translational Modifications", 
J.O. Eidahl, M.E. Hoover, O.E. Branson, L. Zhang, M. Freitas, S.Q. Harper. 
2017 Muscular Dystrophy Association Scientific Conference, Arlington, VA, March 2017.

"Mir-675 reduces DUX4 expression and confers resistance to DUX4 toxicity in FSHD myoblasts: a framework to define the DUX4-targeted miRNome",
N.Y. Saad, S. Garwick-Coppens, S.Q. Harper. 
2017 Muscular Dystrophy Association Scientific Conference, Arlington, VA, March 2017.

"A personalized gene therapy approach for CMT2D",
K.H. Morelli, N. Pyne, A. Fowler, J.S. Domire, S.Q. Harper, and R.W. Burgess. 
2017 Muscular Dystrophy Association Scientific Conference, Arlington, VA, March 2017.

"Developing the first mouse model of FSHD that recapitulates myopathy-related phenotypes",
C. Giesige, K.N. Heller, L.M. Wallace, J.S. Domire, D. Mukweyi, J.O. Eidahl, S.E. Garwick-Coppens, S.M. Guckes, L.R. Rodino-Klapac, and S.Q. Harper. 
Association for Clinical and Translational Science, Washington, D.C., April 2017.

"Developing models and therapies for FSHD", 
S.Q. Harper. 
University of Kansas, Department of Neurology and Neurosurgery, Grand Rounds, Kansas City, MO, August 18, 2017.

"Genome-wide CRISPR screen to identify DUX4 modulating pathways", 
A. Lek, Y. Zhang, M. Lek, J. Criscione, A. Pakula, O. King, P. Jones, L. Kunkel. 
4th Ottawa International conference on neuromuscular disease and biology, Ottawa Canada, September 2017.

"Biomarkers and therapeutic models of FSHD", 
C. P. Emerson and K. Wagner. 
Syros Pharmaceutical, Cambridge, MA, September 6, 2017.

"Emerging molecular therapies for FSHD",
S.Q. Harper. 
FSHD Global Science Week, Patient Outreach Tour of New Zealand and Australia. September 14-21, 2017.

"Emerging molecular therapies for FSHD", 
S.Q. Harper. 
FSHD Global Science Week, Global Science Week, Grand Rounds, Auckland City Hospital, Department of Neurology, Auckland, New Zealand. September 17, 2017.

"RNAi therapy for FSHD", 
S.Q. Harper. 
FSHD Global Scientific Advisory Board, Sydney, Australia, September 18, 2017.

"Developing models and molecular therapies for FSHD", 
S.Q. Harper. 
Walter and Elisa Hall Institute of Medical Research (WEHI) and University of Melbourne, Melbourne, Australia, September 22, 2017.

"Generation of a new, inducible model of FSHD that develops overt myopathic phenotypes", 
C. Giesige, K.N. Heller, L.M. Wallace, J.S. Domire, D. Mukweyi, J.O. Eidahl, S.E. Garwick-Coppens, S.M. Guckes, L.R. Rodino-Klapac, and S.Q. Harper. 
Word Muscle Society, St. Malo, France, October 2017.

"Transcriptome heterogeneity in low-passage myogenic cells from individuals with facioscapulohumeral muscular dystrophy"
L. Hayward, J. Chen, R. Maehr, CP Emerson Jr.
Single Cell Analysis, Cold Spring Harbor, NY, November 8-11, 2017.

"Transcriptome heterogeneity in low passage myogenic cells from individuals with facioscapulohumeral muscular dystrophy",
L. Hayward, J. Chen, R. Maehr, CP Emerson Jr.
Single Cell Analysis, Cold Springs Harbor, NY, September 8-11, 2017

"Understanding pathogenesis and developing therapies for FSHD",
S.Q. Harper.
University of Missouri, Department of Molecular Microbiology and Immunology, Columbia, MO, February 23, 2016.

"The use of humanized mouse models to validate disease association of a de novo GARS variant and to test a novel gene therapy strategy for CMT2D",
K.H. Morelli, J.S. Domire, N. Pyne, S.Q. Harper, and R.W. Burgess. 
Human Genome meeting, Houston, TX, February 28-March 2, 2016.

"DUX4-targeted RNAi therapy for FSHD",
S.Q. Harper.
Friends of FSH Research Summit, Portland, OR, February 29-March 1, 2016.

"Facioscapulohumeral Dystrophy (FSHD)",
L. Kunkel.
Biogen, Cambridge, MA, March 9, 2016.

"Using genome-wide CRISPR libraries to identify modifiers of FSHD",
A. Lek, M. Lek, J. Criscione, A. Pakula, T. Zhang, O. King, P. Jones, L. Kunkel.
Hyatt Regency Crystal City, Arlington, VA, March 19-22, 2016.

"Zebrafish models for FSHD",
A. Pakula, A. Lek, H. Mitsuhashi, F. Rahimov, V. Gupta, J. Criscione, L. Mead, L. Kunkel.
Hyatt Regency Crystal City, Arlington, VA, March 19-22, 2016.

"Translating FSHD",
S.Q. Harper.
Children's Hospital of Pennsylvania/University of Pennsylvania (CHOP/UPENN) Gene Therapy and Vaccines section, Philadelphia, PA, April 4, 2016.

"iPSC modeling of muscle development and disease",
C.P. Emerson.
UT Southwestern, Dallas, TX, April 28, 16.

"Toxicology for DUX4-targeted microRNAs",
L.M. Wallace, D. Griffin, N. Pyne, J.S. Domire, L. Rodino-Klapac, and S.Q. Harper.
American Society of Gene and Cell Therapy Annual Meeting, Washington, D.C. June, 2016.

"RNAI therapy for dominant LGMD1A", 
L.M. Wallace, C.R. Giesiger, D.A. Griffin, L.R. Rodino-Klapac, and S.Q. Harper. 
American Society of Gene and Cell Therapy Annual Meeting, Washington, D.C., June, 2016.

"iPSC modeling of human myogenesis and muscle disease",
M.J. Shi, J.C. Chen, E. Rozsahegyi, C. Spencer, J. Yan, H. Zhou, L. Caron, A. Kiselyov, U. Schmidt, A. Bang, K.R. Wagner, C.P. Emerson, Jr. 
Molecular Mechanisms Modulating Skeletal Muscle Development and Homeostasis in Health and Disease", Asilomar Conference Grounds, Pacific Grove, CA, June 6-11, 2016.

"Genome-wide screen to identify genetic modifiers of FSHD", 
A. Lek, M. l Lek, O. King, P. Jones, L. Kunkel. 
New Directions in Biology and Disease of Skeletal Muscle Meeting, Renaissance Orlando at SeaWorld, FL, June 26-July 02, 2016.

"DUX4 inducible zebrafish model for high-throughput drug screen", 
A. Pakula, A. Lek, H. Mitsuhashi, F. Rahimov, V. Gupta, J. Criscione, L. Mead, L. Kunkel 
New Directions in Biology and Disease of Skeletal Muscle Meeting, Renaissance Orlando at SeaWorld, FL, June 26-July 02, 2016.

"DUX4 inhibition as a therapeutic strategy for FSHD", 
S.Q. Harper.
New Directions in Skeletal Muscle Biology Meeting, Orlando at SeaWorld, FL, June 29-July 2, 2016.

"iPSC induction of myogenic progenitors for human myogenesis and disease studies", 
C.P. Emerson, Jr., M.J. Shi, J.C. Chen, J. Yan, E. Rozsahegyi, L. Caron, A. Kiselyov, U. Schmidt, K.R. Wagner, H. Zhou, and A. Bang. 
FASEB Skeletal Muscle Satellite Cells and Regeneration, Keystone Resort Conference Center, Dillon, CO, July 24-29, 2016

"Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4",
J.O. Eidahl, C.R. Giesige1,J.S. Domire, L.M. Wallace, A. M. Fowler, S.M. Guckes, S.E. Garwick-Coppens, P. Labhart, S.Q. Harper. 
FSHD Wellstone Meeting, Worcester, MA, September 2016.

"A transgenic zebrafish model for FSHD", 
A. Pakula, A. Lek, H. Mitsuhashi, F. Rahimov, V. Gupta, J. Criscione, L. Mead, L. Kunkel. 
FSHD Wellstone Meeting, Worcester, MA, September 2016.

"iPSC-derived myogenic progenitors for studies of human myogenesis and disease modeling", 
C.P. Emerson, Jr., M.J. Shi, J.C. Chen, J. Yan, E. Rozsahegyi, L. Caron, A. Kiselyov, U. Schmidt, K.R. Wagner, H. Zhou, and A. Bang. 
From Stem Cells to Human Development Meeting, Southbridge Hotel & Conference Center, Southbridge, MA, September 25-28, 2016.

“Third Generation Gene Silencing Oligonucleotide Therapeutics for FSHD”,
J.C. Chen, J. Yan, L. Bhagat, W. Jiang, S. Agrawal, K.R. Wagner, and C.P. Emerson, Jr
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA,  November 10-11, 2016.

"Physiological Characterization of Muscles From FSHD Patients With Early Stage Disease",
J. Udaka T. Kitizawa, O. King, J. Leszyk, K. Wagner, and C. P. Emerson, Jr.
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA, November 10-11 2016.

"Translating DUX4-targeted RNAi therapy for FSHD",
L. M. Wallace, D. Griffin, N. Pyne, J.S. Domire, L. Rodino-Klapac, and S.Q. Harper.
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA, November 10-11, 2016.

"Genome-wide screen to identify genetic modifiers of FSHD",
A. Lek, M. I. Lek, O. King, P. Jones, L. Kunkel.
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA, November 10-11, 2016.

"C1QBP Ihibits DUX4-dependent gene activation and can be targeted with 4MU",
W. E. Wright, J. Leszy, and C. P. Emerson Jr.
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA, November 10-11, 2016.

"miR-675 reduces DUX4 expression and confers resistance to DUX4 toxicity in FSHD myoblasts: a framework to define the DUX4-targeted miRNome".
N. Saad, s. Garwick-Coppens, and S.Q. Harper.
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA, November 10-11, 2016.

"Bone health in Facioscapulohumeral muscular dystrophy: a cross-sectional study",
H. Chagarlamudi, A Corbett, M. Stoll, G. Bibat, C. Grosmann, C. Matichack, N. Stinson, J. Shapiro, K.R. Wagner.
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA, November 10-11, 2016.

C1QBP Ihibits DUX4-dependent gene activation and can be targeted with 4MU",
A. M. DeSimone, G. Bibat, K.R. Wagner, G. Stadler, W. E. Wright, J. Leszy, and C. P. Emerson Jr.
FSH Society International Research Consortium & Research Planning Meeting, Westin Hotel, Boston, MA, November 10-11, 2016.

"DUX4 inhibition as a therapeutic strategy for FSHD",
S.Q. Harper.  IGBMC, Strasbourg, France, September 2015.

"DUX4-targeting antisense therapy development in a human muscle xenograft model for FSHD",
T. Zhang.  FSHD International Research Consortium & Research Planning Meeting, Boston, MA, October 5-6, 2015.

"DUX4-targeting antisense therapy development in a human muscle xenograft model for FSHD",
A.M. DeSimone, G. Bibat, K. Wagner, W.E. Stadler, T. Zhang.  FSHD International Research
Consortium & Research Planning Meeting, Boston, MA, October 5-6, 2015

"Toxicology for DUX4-targeted microRNAs",
L.M. Wallace, J.S. Domire, C.R. Giesige, and S.Q. Harper.
FSH Society International Research Consortium & Research Planning Meeting, Boston, MA, October 5-6, 2015.

"The Translocation of DUX4 and DUX4c during myoblast differentiation allows their association with nucleo-cytoplasmic proteins
associated with mRNP granules",
E. Ansseau, J.O. Eidahl, C. Lancelot, A. Tassin, C. Matteotti, C. Yip,, J. Liu, B. Leroy, C.
Hubeau, C. Gerbaux. S. Cloet, A Wauters, S. Zorbo, P. Meyer, I. Pirson, D. Laoudj-Chenivesse,
R. Wattiez, S.Q. Harper, A. Belayew, and F. Coppee. FSH Society International Research Consortium & Research Planning Meeting, Boston, MA, October 5-6, 2015

"Defining the functional overlap between mouse Dux and human DUX4",
J.O. Eidahl, C.R. Giesige, J.S. Domire, L.M. Wallace, A. Fowler, S. Guckes, and S.Q. Harper.
FSH Society International Research Consortium & Research Planning Meeting, Boston, MA, October 5-6, 2015.

"Protein chemistry and protein-protein interactions of DUX4",
J. O. Eidahl and S.Q. Harper.
FSH Society International Research Consortium & Research Planning Meeting, Boston, MA, October 5-6, 2015.

"DUX4 inducible zebrafish model for high-throughput drug screen" ,
A.Pakula, A.Lek, H. Mitsuhashi, F. Rahimov, V. Gupta, J. Criscione, L. Mead, L. Kunkel.
FSH Society International Research Consortium & Research Planning Meeting, Boston, MA, October 5-6, 2015.