Research Output per year
Research Output 1996 2019
- 1 - 50 out of 170 results
- Publication Year, Title (descending)
A homozygous frameshift variant in the KRT5 gene is compatible with life and results in severe recessive epidermolysis bullosa simplex
Tryon, R. K., Tolar, J., Preusser, S. M., Riddle, M. J., Keene, D. R., Bower, M., Thyagarajan, B. & Ebens, C., Jul 1 2019, In : JAAD Case Reports. 5, 7, p. 576-579 4 p.Research output: Contribution to journal › Article
Block Polymer Micelles Enable CRISPR/Cas9 Ribonucleoprotein Delivery: Physicochemical Properties Affect Packaging Mechanisms and Gene Editing Efficiency
Tan, Z., Jiang, Y., Ganewatta, M. S., Kumar, R., Keith, A., Twaroski, K., Pengo, T., Tolar, J., Lodge, T. P. & Reineke, T. M., Oct 21 2019, In : Macromolecules.Research output: Contribution to journal › Article
Bone marrow transplant with post-transplant cyclophosphamide for recessive dystrophic epidermolysis bullosa expands the related donor pool and permits tolerance of nonhaematopoietic cellular grafts
Ebens, C. L., McGrath, J. A., Tamai, K., Hovnanian, A., Wagner, J. E., Riddle, M. J., Keene, D. R., DeFor, T. E., Tryon, R., Chen, M., Woodley, D. T., Hook, K. & Tolar, J., Jan 1 2019, In : British Journal of Dermatology.Research output: Contribution to journal › Article
Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation
Polgreen, L. E., Lund, T. C., Braunlin, E., Tolar, J., Miller, B. S., Fung, E., Whitley, C. B., Eisengart, J. B., Northrop, E., Rudser, K., Miller, W. P. & Orchard, P. J., Jan 1 2019, (Accepted/In press) In : Pediatric Research.Research output: Contribution to journal › Article
Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
Eisengart, J. B., Pierpont, E. I., Kaizer, A. M., Rudser, K. D., King, K. E., Pasquali, M., Polgreen, L. E., Dickson, P. I., Le, S. Q., Miller, W. P., Tolar, J., Orchard, P. J. & Lund, T. C., Nov 1 2019, In : Genetics in Medicine. 21, 11, p. 2552-2560 9 p.Research output: Contribution to journal › Article
Molecular Additives Significantly Enhance Glycopolymer-Mediated Transfection of Large Plasmids and Functional CRISPR-Cas9 Transcription Activation Ex Vivo in Primary Human Fibroblasts and Induced Pluripotent Stem Cells
Boyle, W. S., Twaroski, K., Woska, E. C., Tolar, J. & Reineke, T. M., Feb 20 2019, In : Bioconjugate Chemistry. 30, 2, p. 418-431 14 p.Research output: Contribution to journal › Article
Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes
Lund, T. C., Miller, W. P., Liao, A. Y., Tolar, J., Shanley, R. M., Pasquali, M., Sando, N., Bigger, B. W., Polgreen, L. E. & Orchard, P. J., Dec 1 2019, In : Scientific reports. 9, 1, 14105.Research output: Contribution to journal › Article
Revertant mosaic fibroblasts in recessive dystrophic epidermolysis bullosa
Twaroski, K., Eide, C., Riddle, M. J., Xia, L., Lees, C. J., Chen, W., Mathews, W., Keene, D. R., McGrath, J. A. & Tolar, J., Jan 1 2019, In : British Journal of Dermatology.Research output: Contribution to journal › Article
Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa
Lwin, S. M., Syed, F., Di, W. L., Kadiyirire, T., Liu, L., Guy, A., Petrova, A., Abdul-Wahab, A., Reid, F., Phillips, R., Elstad, M., Georgiadis, C., Aristodemou, S., Lovell, P. A., McMillan, J. R., Mee, J., Miskinyte, S., Titeux, M., Ozoemena, L., Pramanik, R. & 21 others, , Jun 6 2019, In : JCI Insight. 4, 11, :e126243.Research output: Contribution to journal › Article
TCIRG1 Transgenic Rescue of Osteoclast Function Using Induced Pluripotent Stem Cells Derived from Patients with Infantile Malignant Autosomal Recessive Osteopetrosis
Chen, W., Twaroski, K., Eide, C., Riddle, M. J., Orchard, P. J. & Tolar, J., Nov 6 2019, In : The Journal of bone and joint surgery. American volume. 101, 21, p. 1939-1947 9 p.Research output: Contribution to journal › Article
Ternary Composite Nanofibers Containing Chondroitin Sulfate Scavenge Inflammatory Chemokines from Solution and Prohibit Squamous Cell Carcinoma Migration
Boyle, W. S., Chen, W., Rodriguez, A., Linn, S., Tolar, J., Lozano, K. & Reineke, T. M., Feb 18 2019, In : ACS Applied Bio Materials. 2, 2, p. 619-624 6 p.Research output: Contribution to journal › Article
3D Printed Functional and Biological Materials on Moving Freeform Surfaces
Zhu, Z., Guo, S., Hirdler, T., Eide, C. R., Fan, X., Tolar, J. & McAlpine, M., Jun 6 2018, In : Advanced Materials. 30, 23, 1707495.Research output: Contribution to journal › Article
A 3D in vitro model of the dermoepidermal junction amenable to mechanical testing
Jung, J. P., Lin, W. H., Riddle, M. J., Tolar, J. & Ogle, B. M., Dec 2018, In : Journal of Biomedical Materials Research - Part A. 106, 12, p. 3231-3238 8 p.Research output: Contribution to journal › Article
A multitask clustering approach for single-cell RNA-seq analysis in Recessive Dystrophic Epidermolysis Bullosa
Zhang, H., Lee, C. A. A., Li, Z., Garbe, J. R., Eide, C. R., Petegrosso, R., Kuang, R. & Tolar, J., Apr 2018, In : PLoS Computational Biology. 14, 4, e1006053.Research output: Contribution to journal › Article
ATXN1-CIC Complex Is the Primary Driver of Cerebellar Pathology in Spinocerebellar Ataxia Type 1 through a Gain-of-Function Mechanism
Rousseaux, M. W. C., Tschumperlin, T., Lu, H. C., Lackey, E. P., Bondar, V. V., Wan, Y. W., Tan, Q., Adamski, C. J., Friedrich, J., Twaroski, K., Chen, W., Tolar, J., Henzler, C., Sharma, A., Bajić, A., Lin, T., Duvick, L., Liu, Z., Sillitoe, R. V., Zoghbi, H. Y. & 1 others, , Mar 21 2018, In : Neuron. 97, 6, p. 1235-1243.e5Research output: Contribution to journal › Article
Continuous treatment with IL-15 exhausts human NK cells via a metabolic defect
Felices, M., Lenvik, A. J., Mc Elmurry, R. T., Chu, S., Hinderlie, P., Bendzick, L., Geller, M. A., Tolar, J., Blazar, B. R. & Miller, J. S., Feb 8 2018, In : JCI Insight. 3, 3Research output: Contribution to journal › Article
Crispr/cas9-based cellular engineering for targeted gene overexpression
Osborn, M. J., Lees, C. J., McElroy, A. N., Merkel, S. C., Eide, C. R., Mathews, W., Feser, C. J., Tschann, M., McElmury, R. T., Webber, B. R., Kim, C. J., Blazar, B. R. & Tolar, J., Apr 2018, In : International journal of molecular sciences. 19, 4, 946.Research output: Contribution to journal › Article
Human CD19-Targeted Mouse T Cells Induce B Cell Aplasia and Toxicity in Human CD19 Transgenic Mice
Pennell, C. A., Barnum, J. L., McDonald-Hyman, C. S., Panoskaltsis-Mortari, A., Riddle, M. J., Xiong, Z., Loschi, M., Thangavelu, G., Campbell, H. M., Storlie, M. D., Refaeli, Y., Furlan, S. N., Jensen, M. C., Kean, L. S., Miller, J. S., Tolar, J., Osborn, M. J. & Blazar, B. R., Jun 6 2018, In : Molecular Therapy. 26, 6, p. 1423-1434 12 p.Research output: Contribution to journal › Article
Modeling and rescue of defective blood-brain barrier function of induced brain microvascular endothelial cells from childhood cerebral adrenoleukodystrophy patients
Lee, C. A. A., Seo, H. S., Armien, A. G., Bates, F. S., Tolar, J. & Azarin, S. M., Apr 4 2018, In : Fluids and Barriers of the CNS. 15, 1, 9.Research output: Contribution to journal › Article
Montagna Symposium 2017—Precision Dermatology: Next Generation Prevention, Diagnosis, and Treatment
Tolar, J., Bauer, J. W., Kaplan, D. H., Leachman, S. A., McGrath, J. A., Paller, A. S., Griffith-Bauer, K. A., Stemwedel, C. E. & Kulesz-Martin, M. F., Jun 2018, In : Journal of Investigative Dermatology. 138, 6, p. 1243-1248 6 p.Research output: Contribution to journal › Article
Murine chronic graft-versus-host disease proteome profiling discovers CCL15 as a novel biomarker in patients
Du, J., Flynn, R., Paz, K., Ren, H. G., Ogata, Y., Zhang, Q., Gafken, P. R., Storer, B. E., Roy, N. H., Burkhardt, J. K., Mathews, W., Tolar, J., Lee, S. J., Blazar, B. R. & Paczesny, S., Apr 12 2018, In : Blood. 131, 15, p. 1743-1754 12 p.Research output: Contribution to journal › Article
Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita
Fioredda, F., Iacobelli, S., Korthof, E. T., Knol, C., van Biezen, A., Bresters, D., Veys, P., Yoshimi, A., Fagioli, F., Mats, B., Zecca, M., Faraci, M., Miano, M., Arcuri, L., Maschan, M., O'Brien, T., Diaz, M. A., Sevilla, J., Smith, O., Peffault de Latour, R. & 13 others, , Oct 2018, In : British Journal of Haematology. 183, 1, p. 110-118 9 p.Research output: Contribution to journal › Article
Regenerative Solutions for Inherited Diseases
Tolar, J., May 1 2018, In : Clinical pharmacology and therapeutics. 103, 5, p. 763-766 4 p.Research output: Contribution to journal › Article
Reliability and validity of the instrument for scoring clinical outcomes of research for epidermolysis bullosa (iscorEB)
Bruckner, A. L., Fairclough, D. L., Feinstein, J. A., Lara-Corrales, I., Lucky, A. W., Tolar, J. & Pope, E., May 1 2018, In : British Journal of Dermatology. 178, 5, p. 1128-1134 7 p.Research output: Contribution to journal › Article
Thymic epithelial cell support of thymopoiesis does not require Klotho
Xing, Y., Smith, M. J., Goetz, C. A., Mc Elmurry, R. T., Parker, S. L., Min, D., Hollander, G. A., Weinberg, K. I., Tolar, J., Stefanski, H. E. & Blazar, B. R., Dec 1 2018, In : Journal of Immunology. 201, 11, p. 3320-3328 9 p.Research output: Contribution to journal › Article
Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: Potential for biomarker development and insight into pathogenesis
Hinderer, C., Katz, N., Louboutin, J. P., Bell, P., Tolar, J., Orchard, P. J., Lund, T. C., Nayal, M., Weng, L., Mesaros, C., de Souza, C. F. M., Corte, A. D., Giugliani, R. & Wilson, J. M., Oct 1 2017, In : Human molecular genetics. 26, 19, p. 3837-3849 13 p.Research output: Contribution to journal › Article
A Functional Bioluminescent Zebrafish Screen for Enhancing Hematopoietic Cell Homing
Astuti, Y., Kramer, A. C., Blake, A. L., Blazar, B. R., Tolar, J., Taisto, M. E. & Lund, T. C., Jan 10 2017, In : Stem Cell Reports. 8, 1, p. 177-190 14 p.Research output: Contribution to journal › Article
Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology
Osborn, M. J., Webber, B. R., Mc Elmurry, R. T., Rudser, K., DeFeo, A. P., Muradian, M., Petryk, A., Hallgrimsson, B., Blazar, B. R., Tolar, J. & Braunlin, E. A., Mar 1 2017, In : Journal of Inherited Metabolic Disease. 40, 2, p. 281-289 9 p.Research output: Contribution to journal › Article
CRISPR/Cas9-mediated correction of the FANCD1 Gene in primary patient cells
Skvarova Kramarzova, K., Osborn, M. J., Webber, B. R., Defeo, A. P., McElroy, A. N., Kim, C. J. & Tolar, J., Jun 14 2017, In : International journal of molecular sciences. 18, 6, 1269.Research output: Contribution to journal › Article
Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation
Dietz, A. C., Mehta, P. A., Vlachos, A., Savage, S. A., Bresters, D., Tolar, J., Boulad, F., Dalle, J. H., Bonfim, C., de la Fuente, J., Duncan, C. N., Baker, K. S., Pulsipher, M. A., Lipton, J. M., Wagner, J. E. & Alter, B. P., May 1 2017, In : Biology of Blood and Marrow Transplantation. 23, 5, p. 726-735 10 p.Research output: Contribution to journal › Article
Genome-wide Specificity of Highly Efficient TALENs and CRISPR/Cas9 for T Cell Receptor Modification
Knipping, F., Osborn, M. J., Petri, K., Tolar, J., Glimm, H., von Kalle, C., Schmidt, M. & Gabriel, R., Mar 17 2017, In : Molecular Therapy - Methods and Clinical Development. 4, p. 213-224 12 p.Research output: Contribution to journal › Article
Heparin Enhances Transfection in Concert with a Trehalose-Based Polycation with Challenging Cell Types
Boyle, W. S., Senger, K., Tolar, J. & Reineke, T. M., Jan 9 2017, In : Biomacromolecules. 18, 1, p. 56-67 12 p.Research output: Contribution to journal › Article
Large Intragenic Deletion in DSTYK Underlies Autosomal-Recessive Complicated Spastic Paraparesis, SPG23
Lee, J. Y. W., Hsu, C. K., Michael, M., Nanda, A., Liu, L., McMillan, J. R., Pourreyron, C., Takeichi, T., Tolar, J., Reid, E., Hayday, T., Blumen, S. C., Abu-Mouch, S., Straussberg, R., Basel-Vanagaite, L., Barhum, Y., Zouabi, Y., Al-Ajmi, H., Huang, H. Y., Lin, T. C. & 6 others, , Feb 2 2017, In : American Journal of Human Genetics. 100, 2, p. 364-370 7 p.Research output: Contribution to journal › Article
Late Effects Screening Guidelines after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects After Pediatric HCT
Dietz, A. C., Savage, S. A., Vlachos, A., Mehta, P. A., Bresters, D., Tolar, J., Bonfim, C., Dalle, J. H., de la Fuente, J., Skinner, R., Boulad, F., Duncan, C. N., Baker, K. S., Pulsipher, M. A., Lipton, J. M., Wagner, J. E. & Alter, B. P., Sep 1 2017, In : Biology of Blood and Marrow Transplantation. 23, 9, p. 1422-1428 7 p.Research output: Contribution to journal › Article
Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: A prospective study and review of the literature
Mallhi, K., Orchard, P. J., Miller, W. P., Cao, Q., Tolar, J. & Lund, T. C., May 1 2017, In : Bone marrow transplantation. 52, 5, p. 726-732 7 p.Research output: Contribution to journal › Article
PAX7 Targets, CD54, Integrin α9β1, and SDC2, Allow Isolation of Human ESC/iPSC-Derived Myogenic Progenitors
Magli, A., Incitti, T., Kiley, J., Swanson, S. A., Darabi, R., Rinaldi, F., Selvaraj, S., Yamamoto, A., Tolar, J., Yuan, C., Stewart, R., Thomson, J. A. & Perlingeiro, R. C. R., Jun 27 2017, In : Cell reports. 19, 13, p. 2867-2877 11 p.Research output: Contribution to journal › Article
Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients
Ivaturi, V., Dvorak, C. C., Chan, D., Liu, T., Cowan, M. J., Wahlstrom, J., Stricherz, M., Jennissen, C., Orchard, P. J., Tolar, J., Pai, S. Y., Huang, L., Aweeka, F. & Long-Boyle, J., Oct 1 2017, In : Biology of Blood and Marrow Transplantation. 23, 10, p. 1701-1713 13 p.Research output: Contribution to journal › Article
Rapid DNA replication origin licensing protects stem cell pluripotency
Matson, J. P., Dumitru, R., Coryell, P., Baxley, R. M., Chen, W., Twaroski, K., Webber, B. R., Tolar, J., Bielinsky, A. K., Purvis, J. E. & Cook, J. G., Nov 17 2017, In : eLife. 6, e30473.Research output: Contribution to journal › Article
Rapid generation of Col7a1 mouse model of recessive dystrophic epidermolysis bullosa and partial rescue via immunosuppressive dermal mesenchymal stem cells
Webber, B. R., O'Connor, K. T., Mc Elmurry, R. T., Durgin, E. N., Eide, C. R., Lees, C. J., Riddle, M. J., Mathews, W. E., Frank, N. Y., Kluth, M. A., Ganss, C., Moriarity, B. S., Frank, M. H., Osborn, M. J. & Tolar, J., Oct 1 2017, In : Laboratory Investigation. 97, 10, p. 1218-1224 7 p.Research output: Contribution to journal › Article
T cell progenitor therapy-facilitated thymopoiesis depends upon thymic input and continued thymic microenvironment interaction
Smith, M. J., Reichenbach, D. K., Parker, S. L., Riddle, M. J., Mitchell, J., Osum, K. C., Mohtashami, M., Stefanski, H. E., Fife, B. T., Bhandoola, A., Hogquist, K. A., Holländer, G. A., Zúñiga-Pflücker, J. C., Tolar, J. & Blazar, B. R., May 18 2017, In : JCI Insight. 2, 10Research output: Contribution to journal › Article
TP53 Modulates Oxidative Stress in Gata1+ Erythroid Cells
Kramer, A. C., Weber, J., Zhang, Y., Tolar, J., Gibbens, Y. Y., Shevik, M. & Lund, T. C., Feb 14 2017, In : Stem Cell Reports. 8, 2, p. 360-372 13 p.Research output: Contribution to journal › Article
A novel explanation of corneal clouding in a bone marrow transplant-treated patient with Hurler syndrome
Yuan, C., Bothun, E. D., Hardten, D. R., Tolar, J. & McLoon, L. K., Jul 1 2016, In : Experimental Eye Research. 148, p. 83-89 7 p.Research output: Contribution to journal › Article
Cerebral Spinal Fluid levels of Cytokines are elevated in Patients with Metachromatic Leukodystrophy
Thibert, K. A., Raymond, G. V., Tolar, J., Miller, W. P., Orchard, P. J. & Lund, T. C., Apr 15 2016, In : Scientific reports. 6, 24579.Research output: Contribution to journal › Article
CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa
Webber, B. R., Osborn, M. J., McElroy, A. N., Twaroski, K., Lonetree, C., Defeo, A. P., Xia, L., Eide, C., Lees, C. J., McElmurry, R. T., Riddle, M. J., Kim, C. J., Patel, D. D., Blazar, B. R. & Tolar, J., Dec 8 2016, In : Regenerative Medicine. 1Research output: Contribution to journal › Article
CRISPR/Cas9 targeted gene editing and cellular engineering in fanconi anemia
Osborn, M. J., Lonetree, C. L., Webber, B. R., Patel, D. D., Dunmire, S., DeFeo, A. P., McElroy, A. N., MacMillan, M. L., Wagner, J. E., Blazar, B. R. & Tolar, J., Oct 15 2016, In : Stem Cells and Development. 25, 20, p. 1591-1603 13 p.Research output: Contribution to journal › Article
Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres
Ghosh, A., Miller, W., Orchard, P. J., Jones, S. A., Mercer, J., Church, H. J., Tylee, K., Lund, T., Bigger, B. W., Tolar, J. & Wynn, R. F., Mar 1 2016, In : Molecular Genetics and Metabolism. 117, 3, p. 373-377 5 p.Research output: Contribution to journal › Article
Evaluation of TCR gene editing achieved by TALENs, CRISPR/Cas9, and megaTAL nucleases
Osborn, M. J., Webber, B. R., Knipping, F., Lonetree, C. L., Tennis, N., DeFeo, A. P., McElroy, A. N., Starker, C. G., Lee, C., Merkel, S., Lund, T. C., Kelly-Spratt, K. S., Jensen, M. C., Voytas, D. F., Von Kalle, C., Schmidt, M., Gabriel, R., Hippen, K. L., Miller, J. S., Scharenberg, A. M. & 2 others, , Mar 1 2016, In : Molecular Therapy. 24, 3, p. 570-581 12 p.Research output: Contribution to journal › Article
Fast, Efficient, and Gentle Transfection of Human Adherent Cells in Suspension
Agrawal, P., Ingle, N. P., Boyle, W. S., Ward, E., Tolar, J., Dorfman, K. D. & Reineke, T. M., Apr 27 2016, In : ACS Applied Materials and Interfaces. 8, 14, p. 8870-8874 5 p.Research output: Contribution to journal › Article
GSK3β inhibition activates the CDX/HOX pathway and promotes hemogenic endothelial progenitor differentiation from human pluripotent stem cells
Kitajima, K., Nakajima, M., Kanokoda, M., Kyba, M., Dandapat, A., Tolar, J., Saito, M. K., Toyoda, M., Umezawa, A. & Hara, T., Jan 1 2016, In : Experimental Hematology. 44, 1, p. 68-74.e10Research output: Contribution to journal › Article
IL15 Trispecific Killer Engagers (TriKE) Make Natural Killer Cells Specific to CD33+ Targets while Also Inducing Persistence, in Vivo Expansion, and Enhanced Function
Vallera, D. A., Felices, M., McElmurry, R., McCullar, V., Zhou, X., Schmohl, J. U., Zhang, B., Lenvik, A. J., Panoskaltsis-Mortari, A., Verneris, M. R., Tolar, J., Cooley, S., Weisdorf, D. J., Blazar, B. R. & Miller, J. S., Jul 15 2016, In : Clinical Cancer Research. 22, 14, p. 3440-3450 11 p.Research output: Contribution to journal › Article