Jakub Tolar &ndash; Research Output

2019

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

Open Access

Epidermolysis Bullosa Simplex

Keratin-5

Keratins

Genes

Autosomal Recessive Epidermolysis Bullosa Simplex

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

1 Citation (Scopus)

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

Epidermolysis Bullosa Dystrophica

Cyclophosphamide

Bone Marrow

Tissue Donors

Transplants

1 Citation (Scopus)

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

Open Access

Mucopolysaccharidosis I

Cell Transplantation

Clinical Trials

Anti-Idiotypic Antibodies

Pharmaceutical Preparations

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

Open Access

Mucopolysaccharidosis I

Enzyme Replacement Therapy

Biomarkers

Cerebrospinal Fluid

Enzymes

2 Citations (Scopus)

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

Clustered Regularly Interspaced Short Palindromic Repeats

Induced Pluripotent Stem Cells

Transcription

Fibroblasts

Stem cells

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

Open Access

Mucopolysaccharidosis I

Anti-Idiotypic Antibodies

Enzyme Replacement Therapy

Transplants

Glycosaminoglycans

1 Citation (Scopus)

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

Epidermolysis Bullosa Dystrophica

Fibroblasts

Blister

Keratinocytes

Mosaicism

2 Citations (Scopus)

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, Serrano, S., Rowles, R., Maurin, C., Orrin, E., Martinez-Queipo, M., Rashidghamat, E., Tziotzios, C., Onoufriadis, A., Chen, M., Chan, L., Farzaneh, F., Rio, M. D., Tolar, J., Bauer, J. W., Larcher, F., Antoniou, M. N., Hovnanian, A., Thrasher, A. J., Mellerio, J. E., Qasim, W. & McGrath, J. A., Jun 6 2019, In : JCI Insight. 4, 11, :e126243.

Research output: Contribution to journal › Article

Open Access

Epidermolysis Bullosa Dystrophica

Genetic Therapy

Fibroblasts

Safety

Skin

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

Induced Pluripotent Stem Cells

Osteoclasts

Bone Remodeling

Cathepsin K

Osteopetrosis

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

Epidermolysis Bullosa Dystrophica

Nanofibers

Chondroitin Sulfates

Chemokines

Cell Movement

2018

22 Citations (Scopus)

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

Open Access

Functional materials

Biological materials

Printing

Hydrogels

Real time control

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

Open Access

Mechanical testing

Fibroblasts

Collagen Type VII

Adhesion

Collagen

4 Citations (Scopus)

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

Open Access

epidermolysis bullosa

Epidermolysis Bullosa Dystrophica

RNA

Cluster Analysis

Clustering

7 Citations (Scopus)

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, Orr, H. T., Mar 21 2018, In : Neuron. 97, 6, p. 1235-1243.e5

Research output: Contribution to journal › Article

Spinocerebellar Ataxias

Pathology

Cerebellum

Neurons

Purkinje Cells

20 Citations (Scopus)

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, 3

Research output: Contribution to journal › Article

Open Access

Interleukin-15

Natural Killer Cells

Therapeutics

cdc Genes

Fatty Acids

5 Citations (Scopus)

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

Open Access

Cell Engineering

genes

Genes

engineering

T-cells

7 Citations (Scopus)

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

Open Access

Transgenic Mice

B-Lymphocytes

T-Lymphocytes

T-Cell Antigen Receptor

Antigen Receptors

6 Citations (Scopus)

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

Open Access

Adrenoleukodystrophy

Blood-Brain Barrier

Endothelial Cells

Brain

Ethylene Oxide

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

Precision Medicine

Dermatology

Genomics

Skin Diseases

Biomedical Research

6 Citations (Scopus)

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

Open Access

Biomarkers

Graft vs Host Disease

Proteome

Grafts

T-cells

10 Citations (Scopus)

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, de la Fuente, J., Or, R., Van Lint, M. T., Tolar, J., Aljurf, M., Fisher, A., Skorobogatova, E. V., Diaz de Heredia, C., Risitano, A., Dalle, J. H., Sedláček, P., Ghavamzadeh, A. & Dufour, C., Oct 2018, In : British Journal of Haematology. 183, 1, p. 110-118 9 p.

Research output: Contribution to journal › Article

Dyskeratosis Congenita

Hematopoietic Stem Cell Transplantation

Disease-Free Survival

Bone Marrow

Inborn Genetic Diseases

1 Citation (Scopus)

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

Inborn Genetic Diseases

Regenerative Medicine

Computational Biology

Health

Therapeutics

3 Citations (Scopus)

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

Epidermolysis Bullosa

Reproducibility of Results

Outcome Assessment (Health Care)

Quality of Life

Epidermolysis Bullosa Dystrophica

1 Citation (Scopus)

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

Open Access

Vitamin D

Epithelial Cells

Thymus Gland

T-Lymphocytes

Thymocytes

2017

3 Citations (Scopus)

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

Open Access

Mucopolysaccharidoses

Polyamines

Spermine

Mucopolysaccharidosis I

Biomarkers

5 Citations (Scopus)

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

Open Access

Bioluminescence

Zebrafish

Ergosterol

Imaging techniques

Firefly Luciferases

4 Citations (Scopus)

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

Open Access

Iduronidase

Mucopolysaccharidosis I

Angiotensin Receptors

Renin-Angiotensin System

Pathology

9 Citations (Scopus)

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

Clustered Regularly Interspaced Short Palindromic Repeats

anemias

Fanconi Anemia

genes

Genes

13 Citations (Scopus)

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

Open Access

Cell Transplantation

Fanconi Anemia

Bone Marrow

Pediatrics

Transplants

12 Citations (Scopus)

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

Open Access

Clustered Regularly Interspaced Short Palindromic Repeats

T-Cell Antigen Receptor

Genome

Guide RNA

Integrases

4 Citations (Scopus)

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

Trehalose

Heparin

A73025

Fibroblasts

polycations

10 Citations (Scopus)

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, Akiyama, M., Lee, J. Y. Y., McLean, W. H. I., Simpson, M. A., Parsons, M. & McGrath, J. A., Feb 2 2017, In : American Journal of Human Genetics. 100, 2, p. 364-370 7 p.

Research output: Contribution to journal › Article

Open Access

Spastic Paraparesis

Protein-Serine-Threonine Kinases

Protein-Tyrosine Kinases

Pedigree

Keratinocytes

7 Citations (Scopus)

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

Open Access

Cell Transplantation

Bone Marrow

Guidelines

Pediatrics

Transplants

1 Citation (Scopus)

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

Open Access

Cell Transplantation

Prospective Studies

Transplants

Adrenoleukodystrophy

Busulfan

15 Citations (Scopus)

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

Open Access

Stem cells

Integrins

Muscle

Pluripotent Stem Cells

Muscles

6 Citations (Scopus)

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

Open Access

Pharmacokinetics

Pediatrics

Transplants

Cell Transplantation

Therapeutics

15 Citations (Scopus)

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

Open Access

Replication Origin

Licensure

Stem cells

DNA Replication

Stem Cells

6 Citations (Scopus)

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

Open Access

Epidermolysis Bullosa Dystrophica

Immunosuppressive Agents

Mesenchymal Stromal Cells

Skin

Clustered Regularly Interspaced Short Palindromic Repeats

4 Citations (Scopus)

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, 10

Research output: Contribution to journal › Article

Open Access

Cell- and Tissue-Based Therapy

Epithelial Cells

T-Lymphocytes

Thymus Gland

Thymocytes

2 Citations (Scopus)

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

Open Access

Erythroid Cells

Oxidative stress

Oxidative Stress

Reactive Oxygen Species

Zebrafish

2016

2 Citations (Scopus)

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

Mucopolysaccharidosis I

Cornea

Bone Marrow

Transplants

Collagen

3 Citations (Scopus)

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

Metachromatic Leukodystrophy

Cytokines

Sulfoglycosphingolipids

Lysosomal Storage Diseases

Interleukin 1 Receptor Antagonist Protein

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. 1

Research output: Contribution to journal › Article

14 Citations (Scopus)

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

Clustered Regularly Interspaced Short Palindromic Repeats

Cell Engineering

Fanconi Anemia

Induced Pluripotent Stem Cells

Genes

30 Citations (Scopus)

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

Mucopolysaccharidosis I

Enzyme Replacement Therapy

Transplants

Hematopoietic Stem Cell Transplantation

Stem cells

58 Citations (Scopus)

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, Tolar, J. & Blazar, B. R., Mar 1 2016, In : Molecular Therapy. 24, 3, p. 570-581 12 p.

Research output: Contribution to journal › Article

Clustered Regularly Interspaced Short Palindromic Repeats

T-Lymphocytes

T-Cell Antigen Receptor

Adoptive Immunotherapy

Neoplasm Antigens

3 Citations (Scopus)

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

Suspensions

Genes

Cells

Gene transfer

Physiology

13 Citations (Scopus)

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.e10

Research output: Contribution to journal › Article

Induced Pluripotent Stem Cells

Pluripotent Stem Cells

Endothelial Cells

Cell Differentiation

Mesoderm

77 Citations (Scopus)

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

Interleukin-15

Natural Killer Cells

Antigens

Cell Degranulation

Survival