Kathryn Rae Wagner

Professor

1989 …2020

Research output per year

If you made any changes in Pure these will be visible here soon.

Research Output

2020

A decade of optimizing drug development for rare neuromuscular disorders through TACT

Wagner, K. R., De Luca, A., Caizergues, D., Dowling, J., Goemans, N., Gordish-Dressman, H., Grounds, M. D., Kelly, M., Mayhew, A., McNally, E. M., Zoetis, T., Lee, J., Turner, C., Wells, D. J., Csimma, C. & Straub, V., Jan 1 2020, In : Nature Reviews Drug Discovery. 19, 1

Research output: Contribution to journalComment/debate

Open Access

Improving translatability of preclinical studies for neuromuscular disorders: Lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

Willmann, R., Lee, J., Turner, C., Nagaraju, K., Aartsma-Rus, A., Wells, D. J., Wagner, K. R., Csimma, C., Straub, V., Grounds, M. D. & de Luca, A., Jan 1 2020, In : DMM Disease Models and Mechanisms. 13, 2, dmm042903.

Research output: Contribution to journalReview article

Open Access

Stem cell-based therapies for Duchenne muscular dystrophy

Sun, C., Serra, C., Lee, G. & Wagner, K. R., Jan 2020, In : Experimental Neurology. 323, 113086.

Research output: Contribution to journalReview article

2019

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

Nance, M. E., Shi, R., Hakim, C. H., Wasala, N. B., Yue, Y., Pan, X., Zhang, T., Robinson, C. A., Duan, S. X., Yao, G., Yang, N. N., Chen, S. J., Wagner, K. R., Gersbach, C. A. & Duan, D., Jan 1 2019, In : Molecular Therapy.

Research output: Contribution to journalArticle

Benign tumors in myotonic dystrophy type I target disease-related cancer sites

Alsaggaf, R., St. George, D. M. M., Zhan, M., Pfeiffer, R. M., Wang, Y., Anderson, L. A., Liu, Z., Koshiol, J., Bauer, A. J., Wagner, K. R., Greene, M. H., Amr, S. & Gadalla, S. M., Jan 1 2019, In : Annals of Clinical and Translational Neurology. 6, 8, p. 1510-1518 9 p.

Research output: Contribution to journalArticle

Open Access

Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): Protocol of a large, international, multi-center prospective study

Lorusso, S., Johnson, N. E., McDermott, M. P., Eichinger, K., Butterfield, R. J., Carraro, E., Higgs, K., Lewis, L., Mul, K., Sacconi, S., Sansone, V. A., Shieh, P., Van Engelen, B., Wagner, K., Wang, L., Statland, J. M., Tawil, R., Dimachkie, M., Pasnoor, M., Roath, K. & 32 others, McCalley, A., Currence, M., Herbelin, L., Hamel, J., Arnold, W. D., Alexander, T., Yankie, M., Kelly, K., Holmberg, B., Diaz, L., Jones, A., Butler, A., Moldt, S., Wilson, A., McIntyre, M., Leung, D., Bibat, G., Yep, M., Stinson, N., Jaworek, A., Sissons-Ross, L., Johnstone, L., Skura, C., Deguzman, D., Cornelissen, Y., Villa, L., Puma, A., Gambella, M., Shi, Y., Garcia, J., Beshiri, F. & Mauro, L., Sep 10 2019, In : BMC neurology. 19, 1, 224.

Research output: Contribution to journalArticle

Open Access

Diabetes, metformin and cancer risk in myotonic dystrophy type I

Alsaggaf, R., Pfeiffer, R. M., Wang, Y., St. George, D. M. M., Zhan, M., Wagner, K. R., Amr, S., Greene, M. H. & Gadalla, S. M., Jan 1 2019, (Accepted/In press) In : International Journal of Cancer.

Research output: Contribution to journalArticle

Engineering 3D skeletal muscle primed for neuromuscular regeneration following volumetric muscle loss

Gilbert-Honick, J., Wagner, K. R., Mao, H. Q. & Grayson, W. L., Jan 1 2019, Society for Biomaterials Annual Meeting and Exposition 2019: The Pinnacle of Biomaterials Innovation and Excellence - Transactions of the 42nd Annual Meeting. Society for Biomaterials, 1 p. (Transactions of the Annual Meeting of the Society for Biomaterials and the Annual International Biomaterials Symposium; vol. 40).

Research output: Chapter in Book/Report/Conference proceedingConference contribution

Identification of the hyaluronic acid pathway as a therapeutic target for facioscapulohumeral muscular dystrophy

DeSimone, A. M., Leszyk, J., Wagner, K. & Emerson, C. P., Dec 11 2019, In : Science Advances. 5, 12, eaaw7099.

Research output: Contribution to journalArticle

Open Access

Long-read single-molecule maps of the functional methylome

Sharim, H., Grunwald, A., Gabrieli, T., Michaeli, Y., Margalit, S., Torchinsky, D., Arielly, R., Nifker, G., Juhasz, M., Gularek, F., Almalvez, M., Dufault, B., Chandra, S. S., Liu, A., Bhattacharya, S., Chen, Y. W., Vilain, E., Wagner, K. R., Pevsner, J. A., Reifenberger, J. & 6 others, Lam, E. T., Hastie, A. R., Cao, H., Barseghyan, H., Weinhold, E. & Ebenstein, Y., Apr 1 2019, In : Genome Research. 29, 4, p. 646-656 11 p.

Research output: Contribution to journalArticle

Open Access

Mss51 deletion enhances muscle metabolism and glucose homeostasis in mice

Rovira Gonzalez, Y. I., Moyer, A. L., LeTexier, N. J., Bratti, A. D., Feng, S., Sun, C., Liu, T., Mula, J., Jha, P., Iyer, S. R., Lovering, R., O’Rourke, B., Noh, H. L., Suk, S., Kim, J. K., Essien Umanah, G. K. & Wagner, K. R., Sep 17 2019, In : JCI Insight. 4, 20, e122247.

Research output: Contribution to journalArticle

Open Access

Performing Human Skeletal Muscle Xenografts in Immunodeficient Mice

Britson, K. A., Black, A. D., Wagner, K. R. & Lloyd, T., Sep 16 2019, In : Journal of visualized experiments : JoVE. 151

Research output: Contribution to journalArticle

The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy

Thangarajh, M., Kaat, A. J., Bibat, G. M., Mansour, J., Summerton, K., Gioia, A., Berger, C., Hardy, K. K. & Wagner, K. R., Jan 1 2019, (Accepted/In press) In : Annals of Clinical and Translational Neurology.

Research output: Contribution to journalArticle

Open Access
2018

Adipose-derived Stem/Stromal Cells on Electrospun Fibrin Microfiber Bundles Enable Moderate Muscle Reconstruction in a Volumetric Muscle Loss Model

Gilbert-Honick, J., Ginn, B., Zhang, Y., Salehi, S., Wagner, K. R., Mao, H. Q. & Grayson, W. L., Nov 1 2018, In : Cell Transplantation. 27, 11, p. 1644-1656 13 p.

Research output: Contribution to journalArticle

A transition toolkit for Duchenne muscular dystrophy

Trout, C. J., Case, L. E., Clemens, P. R., McArthur, A., Noritz, G., Ritzo, M., Wagner, K. R., Vroom, E. & Kennedy, A., Oct 1 2018, In : Pediatrics. 142, p. S110-S117

Research output: Contribution to journalArticle

Biological scaffold-mediated delivery of myostatin inhibitor promotes a regenerative immune response in an animal model of Duchenne muscular dystrophy

Estrellas, K. M., Chung, L., Cheu, L. A., Sadtler, K., Majumdar, S., Mula, J., Wolf, M. T., Elisseeff, J. H. & Wagner, K. R., Jan 1 2018, In : Journal of Biological Chemistry. 293, 40, p. 15594-15605 12 p.

Research output: Contribution to journalArticle

Cardiac management of the patient with Duchenne muscular dystrophy

Buddhe, S., Cripe, L., Friedland-Little, J., Kertesz, N., Eghtesady, P., Finder, J., Hor, K., Judge, D. P., Kinnett, K., McNally, E. M., Raman, S., Thompson, R., Wagner, K. R. & Olson, A. K., Oct 1 2018, In : Pediatrics. 142, p. S72-S81

Research output: Contribution to journalArticle

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

DMD Care Considerations Working Group, Mar 1 2018, In : The Lancet Neurology. 17, 3, p. 211-212 2 p.

Research output: Contribution to journalReview article

Engineering functional and histological regeneration of vascularized skeletal muscle

Gilbert-Honick, J., Iyer, S. R., Somers, S. M., Lovering, R. M., Wagner, K. R., Mao, H. Q. & Grayson, W. L., May 1 2018, In : Biomaterials. 164, p. 70-79 10 p.

Research output: Contribution to journalArticle

Multivoxel proton magnetic resonance spectroscopy in facioscapulohumeral muscular dystrophy

Leung, D. G., Wang, X., Barker, P. B., Carrino, J. A. & Wagner, K. R., Jun 1 2018, In : Muscle and Nerve. 57, 6, p. 958-963 6 p.

Research output: Contribution to journalArticle

Neurology care, diagnostics, and emerging therapies of the patient with Duchenne muscular dystrophy

Leigh, F., Ferlini, A., Biggar, D., Bushby, K., Finkel, R., Morgenroth, L. P. & Wagner, K. R., Oct 1 2018, In : Pediatrics. 142, p. S5-S16

Research output: Contribution to journalArticle

Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

the DEMAND V study group, Aug 1 2018, In : Annals of Clinical and Translational Neurology. 5, 8, p. 913-926 14 p.

Research output: Contribution to journalArticle

2017

Bone health in facioscapulohumeral muscular dystrophy: A cross-sectional study

Chagarlamudi, H., Corbett, A., Stoll, M., Bibat, G. M., Grosmann, C., Matichak Stock, C., Stinson, N., Shapiro, J. & Wagner, K. R., Dec 1 2017, In : Muscle and Nerve. 56, 6, p. 1108-1113 6 p.

Research output: Contribution to journalArticle

Left ventricular dysfunction and conduction disturbances in patients with myotonic muscular dystrophy type I and II

Tanawuttiwat, T., Wagner, K. R., Tomaselli, G. & Nazarian, S., Feb 1 2017, In : JAMA Cardiology. 2, 2, p. 225-228 4 p.

Research output: Contribution to journalArticle

2016

Clinical practice considerations in facioscapulohumeral muscular dystrophy Sydney, Australia, 21 September 2015

Tawil, R., Mah, J. K., Baker, S., Wagner, K. R. & Ryan, M. M., 2016, (Accepted/In press) In : Neuromuscular Disorders.

Research output: Contribution to journalArticle

Clinical trial preparedness in facioscapulohumeral muscular dystrophy: Clinical, tissue, and imaging outcome measures 29-30 May 2015, Rochester, New York

The FSHD Workshop Participants, Feb 1 2016, In : Neuromuscular Disorders. 26, 2, p. 181-186 6 p.

Research output: Contribution to journalArticle

Developing a pro-regenerative biomaterial scaffold microenvironment requires T helper 2 cells

Sadtler, K., Estrellas, K., Allen, B. W., Wolf, M. T., Fan, H., Tam, A. J., Patel, C. H., Luber, B. S., Wang, H., Wagner, K. R., Powell, J. D., Housseau, F., Pardoll, D. M. & Elisseeff, J. H., Apr 15 2016, In : Science. 352, 6283, p. 366-370 5 p.

Research output: Contribution to journalArticle

Dystrophin-deficient dogs with reduced myostatin have unequal muscle growth and greater joint contractures

Kornegay, J. N., Bogan, D. J., Bogan, J. R., Dow, J. L., Wang, J., Fan, Z., Liu, N., Warsing, L. C., Grange, R. W., Ahn, M., Balog-Alvarez, C. J., Cotten, S. W., Willis, M. S., Brinkmeyer-Langford, C., Zhu, H., Palandra, J., Morris, C. A., Styner, M. A. & Wagner, K. R., Apr 4 2016, In : Skeletal Muscle. 6, 1, 14.

Research output: Contribution to journalArticle

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy

Choudhury, S. R., Fitzpatrick, Z., Harris, A. F., Maitland, S. A., Ferreira, J. S., Zhang, Y., Ma, S., Sharma, R. B., Gray-Edwards, H. L., Johnson, J. A., Johnson, A. K., Alonso, L. C., Punzo, C., Wagner, K. R., Maguire, C. A., Kotin, R. M., Martin, D. R. & Sena-Esteves, M., Jun 7 2016, (Accepted/In press) In : Molecular Therapy.

Research output: Contribution to journalArticle

Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics

Chen, J. CJ., King, O. D., Zhang, Y., Clayton, N. P., Spencer, C., Wentworth, B. M., Emerson, C. P. & Wagner, K. R., Jul 5 2016, (Accepted/In press) In : Molecular Therapy.

Research output: Contribution to journalArticle

Validity of the 6 minute walk test in facioscapulohumeral muscular dystrophy

Eichinger, K., Heatwole, C., Heininger, S., Stinson, N., Matichak Stock, C., Grosmann, C., Wagner, K. R., Tawil, R. & Statland, J. M., 2016, (Accepted/In press) In : Muscle and Nerve.

Research output: Contribution to journalArticle

2015

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model

Choi, I. Y., Lim, H. T., Estrellas, K., Mula, J., Cohen, T. V., Zhang, Y., Donnelly, C. J., Richard, J-P., Kim, Y. J., Kim, H., Kazuki, Y., Oshimura, M., Li, H. L., Hotta, A., Rothstein, J. D., Maragakis, N. J., Wagner, K. R. & Lee, G., Sep 9 2015, (Accepted/In press) In : Cell Reports.

Research output: Contribution to journalArticle

Contemporary cardiac issues in Duchenne muscular dystrophy

McNally, E. M., Kaltman, J. R., Woodrow Benson, D., Canter, C. E., Cripe, L. H., Duan, D., Finder, J. D., Hoffman, E. P., Judge, D. P., Kertesz, N., Kinnett, K., Kirsch, R., Metzger, J. M., Pearson, G. D., Rafael-Fortney, J. A., Raman, S. V., Spurney, C. F., Targum, S. L., Wagner, K. R. & Markham, L. W., 2015, In : Circulation. 131, 18, p. 1590-1598 9 p.

Research output: Contribution to journalArticle

Genetic disruption of Smad7 impairs skeletal muscle growth and regeneration

Cohen, T. V., Kollias, H. D., Liu, N., Ward, C. W. & Wagner, K. R., Jun 1 2015, In : Journal of Physiology. 593, 11, p. 2479-2497 19 p.

Research output: Contribution to journalArticle

Individual epigenetic status of the pathogenic D4Z4 macrosatellite correlates with disease in facioscapulohumeral muscular dystrophy

Jones, T. I., King, O. D., Himeda, C. L., Homma, S., Chen, J. C. J., Beermann, M. L., Yan, C., Emerson, C. P., Miller, J. B., Wagner, K. R. & Jones, P. L., 2015, In : Clinical Epigenetics. 7, 1, 37.

Research output: Contribution to journalArticle

Mammalian Mss51 is a Skeletal Muscle-Specific Gene Modulating Cellular Metabolism

Moyer, A. L. & Wagner, K. R., Jan 1 2015, In : Journal of Neuromuscular Diseases. 2, 4, p. 371-385 15 p.

Research output: Contribution to journalArticle

Results of a two-year pilot study of clinical outcome measures in collagen VI- and laminin alpha2-related congenital muscular dystrophies

Meilleur, K. G., Jain, M. S., Hynan, L. S., Shieh, C. Y., Kim, E., Waite, M., McGuire, M., Fiorini, C., Glanzman, A. M., Main, M., Rose, K., Duong, T., Bendixen, R., Linton, M. M., Arveson, I. C., Nichols, C., Yang, K., Fischbeck, K. H., Wagner, K. R., North, K. & 13 others, Mankodi, A., Grunseich, C., Hartnett, E. J., Smith, M., Donkervoort, S., Schindler, A., Kokkinis, A., Leach, M., Foley, A. R., Collins, J., Muntoni, F., Rutkowski, A. & Bönnemann, C. G., Jan 1 2015, In : Neuromuscular Disorders. 25, 1, p. 43-54 12 p.

Research output: Contribution to journalArticle

The TREAT-NMD advisory committee for therapeutics (TACT): An innovative de-risking model to foster orphan drug development

Heslop, E., Csimma, C., Straub, V., McCall, J., Nagaraju, K., Wagner, K. R., Caizergues, D., Korinthenberg, R., Flanigan, K. M., Kaufmann, P., McNeil, E., Mendell, J., Hesterlee, S., Wells, D. J. & Bushby, K., Apr 23 2015, In : Orphanet journal of rare diseases. 10, 1, 49.

Research output: Contribution to journalArticle

Whole-body magnetic resonance imaging evaluation of facioscapulohumeral muscular dystrophy

Leung, D. G., Carrino, J. A., Wagner, K. R. & Jacobs, M., Oct 1 2015, In : Muscle and Nerve. 52, 4, p. 512-520 9 p.

Research output: Contribution to journalArticle

2014

Humanskeletal musclexenograft as anewpreclinical model for muscle disorders

Zhang, Y., King, O. D., Rahimov, F., Jones, T. I., Ward, C. W., Kerr, J. P., Liu, N., Emerson, C. P., Kunkel, L. M., Partridge, T. A. & Wagner, K. R., Jan 1 2014, In : Human molecular genetics. 23, 12, p. 3180-3188 9 p.

Research output: Contribution to journalArticle

Magnetic resonance imaging phenotyping of Becker muscular dystrophy

Faridian-Aragh, N., Wagner, K. R., Leung, D. G. & Carrino, J. A., Dec 1 2014, In : Muscle and Nerve. 50, 6, p. 962-967 6 p.

Research output: Contribution to journalArticle

Novel Mutations Widen the Phenotypic Spectrum of Slow Skeletal/β-Cardiac Myosin (MYH7) Distal Myopathy

Lamont, P. J., Wallefeld, W., Hilton-Jones, D., Udd, B., Argov, Z., Barboi, A. C., Bonneman, C., Boycott, K. M., Bushby, K., Connolly, A. M., Davies, N., Beggs, A. H., Cox, G. F., Dastgir, J., Dechene, E. T., Gooding, R., Jungbluth, H., Muelas, N., Palmio, J., Penttilä, S. & 10 others, Schmedding, E., Suominen, T., Straub, V., Staples, C., Van den Bergh, P. Y. K., Vilchez, J. J., Wagner, K. R., Wheeler, P. G., Wraige, E. & Laing, N. G., 2014, In : Human Mutation. 35, 7, p. 868-879 12 p.

Research output: Contribution to journalArticle

Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy

Leung, D. G., Herzka, D., Thompson, R., He, B., Bibat, G. M., Tennekoon, G., Russell, S. D., Schuleri, K. H., Lardo, A. C., Kass, D. A., Thompson, R., Judge, D. P. & Wagner, K. R., Oct 1 2014, In : Annals of Neurology. 76, 4, p. 541-549 9 p.

Research output: Contribution to journalArticle

Telomere position effect: Regulation of gene expression with progressive telomere shortening over long distances

Robin, J. D., Ludlow, A. T., Batten, K., Magdinier, F., Stadler, G., Wagner, K. R., Shay, J. W. & Wright, W. E., Nov 15 2014, In : Genes & development. 28, 22, p. 2464-2476 13 p.

Research output: Contribution to journalArticle

2013

ANO5 Gene Analysis in a Large Cohort of Patients with Anoctaminopathy: Confirmation of Male Prevalence and High Occurrence of the Common Exon 5 Gene Mutation

Sarkozy, A., Hicks, D., Hudson, J., Laval, S. H., Barresi, R., Hilton-Jones, D., Deschauer, M., Harris, E., Rufibach, L., Hwang, E., Bashir, R., Walter, M. C., Krause, S., van den Bergh, P., Illa, I., Pénisson-Besnier, I., De Waele, L., Turnbull, D., Guglieri, M., Schrank, B. & 43 others, Schoser, B., Seeger, J., Schreiber, H., Gläser, D., Eagle, M., Bailey, G., Walters, R., Longman, C., Norwood, F., Winer, J., Muntoni, F., Hanna, M., Roberts, M., Bindoff, L. A., Brierley, C., Cooper, R. G., Cottrell, D. A., Davies, N. P., Gibson, A., Gorman, G. S., Hammans, S., Jackson, A. P., Khan, A., Lane, R., Mcconville, J., Mcentagart, M., Al-Memar, A., Nixon, J., Panicker, J., Parton, M., Petty, R., Price, C. J., Rakowicz, W., Ray, P., Schapira, A. H., Swingler, R., Turner, C., Wagner, K. R., Maddison, P., Shaw, P. J., Straub, V., Bushby, K. & Lochmüller, H., Aug 2013, In : Human Mutation. 34, 8, p. 1111-1118 8 p.

Research output: Contribution to journalArticle

'Double trouble': Diagnostic challenges in Duchenne muscular dystrophy in patients with an additional hereditary skeletal dysplasia

Donkervoort, S., Schindler, A., Tesi-Rocha, C., Schreiber, A., Leach, M. E., Dastgir, J., Hu, Y., Mankodi, A., Wagner, K. R., Friedman, N. R. & Bönnemann, C. G., Dec 1 2013, In : Neuromuscular Disorders. 23, 12, p. 955-961 7 p.

Research output: Contribution to journalArticle

Fast skeletal muscle troponin activator in the dy2J muscular dystrophy model

Miciak, J. J., Warsing, L. C., Tibbs, M. E., Jasper, J. R., Jampel, S. B., Malik, F. I., Tankersley, C. & Wagner, K. R., Aug 2013, In : Muscle and Nerve. 48, 2, p. 279-285 7 p.

Research output: Contribution to journalArticle

Reevaluating measures of disease progression in facioscapulohumeral muscular dystrophy

Statland, J. M., McDermott, M. P., Heatwole, C., Martens, W. B., Pandya, S., van der Kooi, E. L., Kissel, J. T., Wagner, K. R. & Tawil, R., Apr 2013, In : Neuromuscular Disorders. 23, 4, p. 306-312 7 p.

Research output: Contribution to journalArticle

Telomere position effect regulates DUX4 in human facioscapulohumeral muscular dystrophy

Stadler, G., Rahimov, F., King, O. D., Chen, J. C. J., Robin, J. D., Wagner, K. R., Shay, J. W., Emerson, C. P. & Wright, W. E., Jun 2013, In : Nature structural & molecular biology. 20, 6, p. 671-678 8 p.

Research output: Contribution to journalArticle