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Research Output 2004 2020

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Article
2020

Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs

Osborn, M. J., Newby, G. A., McElroy, A. N., Knipping, F., Nielsen, S. C., Riddle, M. J., Xia, L., Chen, W., Eide, C. R., Webber, B. R., Wandall, H. H., Dabelsteen, S., Blazar, B. R., Liu, D. R. & Tolar, J., Feb 2020, In : Journal of Investigative Dermatology. 140, 2, p. 338-347.e5

Research output: Contribution to journalArticle

Epidermolysis Bullosa Dystrophica
Induced Pluripotent Stem Cells
Adenine
Fibroblasts
Stem cells

Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy

Kim, Y. M., Yum, M. S., Heo, S. H., Kim, T., Jin, H. K., Bae, J. S., Seo, G. H., Oh, A., Yoon, H. M., Lim, H. T., Kim, H. W., Ko, T. S., Lim, H. S., Osborn, M. J., Tolar, J., Cozma, C., Rolfs, A., Zimran, A., Lee, B. H. & Yoo, H. W., Feb 1 2020, In : Journal of medical genetics. 57, 2, p. 124-131 8 p.

Research output: Contribution to journalArticle

Open Access
Ambroxol
Myoclonic Epilepsy
Gaucher Disease
Enzyme Replacement Therapy
Seizures

Phenotype categorization of neurofibromatosis type I and correlation to NF1 mutation types

Kang, E., Kim, Y. M., Seo, G. H., Oh, A., Yoon, H. M., Ra, Y. S., Kim, E. K., Kim, H., Heo, S. H., Kim, G. H., Osborn, M. J., Tolar, J., Yoo, H. W. & Lee, B. H., Jan 1 2020, In : Journal of Human Genetics. 65, 2, p. 79-89 11 p.

Research output: Contribution to journalArticle

Neurofibromatosis 1
Phenotype
Mutation
Neurofibromatosis 1 Genes
Inborn Genetic Diseases
2019

Donor and host B7-H4 expression negatively regulates acute graft-versus-host disease lethality

Saha, A., Taylor, P. A., Lees, C. J., Panoskaltsis-Mortari, A., Osborn, M. J., Feser, C. J., Thangavelu, G., Melchinger, W., Refaeli, Y., Hill, G. R., Munn, D. H., Murphy, W. J., Serody, J. S., Maillard, I., Kreymborg, K., van den Brink, M., Dong, C., Huang, S., Zang, X., Allison, J. P. & 2 others, Zeiser, R. & Blazar, B. R., Jan 1 2019, In : JCI Insight. 4, 19, e127716.

Research output: Contribution to journalArticle

Open Access
Graft vs Host Disease
T-Lymphocytes
Mortality
Metabolic Networks and Pathways
Oxygen Consumption

Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors

Webber, B. R., Lonetree, C. L., Kluesner, M. G., Johnson, M. J., Pomeroy, E. J., Diers, M. D., Lahr, W. S., Draper, G. M., Slipek, N. J., Smeester, B. S., Lovendahl, K. N., McElroy, A. N., Gordon, W. R., Osborn, M. J. & Moriarity, B. S., Dec 1 2019, In : Nature communications. 10, 1, 5222.

Research output: Contribution to journalArticle

Open Access
Cell engineering
Cell Engineering
T-cells
strands
nuclease
2018
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 journalArticle

Open Access
Cell Engineering
genes
Genes
engineering
T-cells
8 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 journalArticle

Open Access
Transgenic Mice
B-Lymphocytes
T-Lymphocytes
T-Cell Antigen Receptor
Antigen Receptors
4 Citations (Scopus)

The vimentin intermediate filament network restrains regulatory T cell suppression of graft-versus-host disease

McDonald-Hyman, C. S., Muller, J. T., Loschi, M., Thangavelu, G., Saha, A., Kumari, S., Reichenbach, D. K., Smith, M. J., Zhang, G., Koehn, B., Lin, J., Mitchell, J. S., Fife, B. T., Panoskaltsis-Mortari, A., Feser, C. J., Kirchmeier, A. K., Osborn, M. J., Hippen, K. L., Kelekar, A., Serody, J. S. & 6 others, Turka, L. A., Munn, D. H., Chi, H., Neubert, T. A., Dustin, M. L. & Blazar, B. R., Oct 1 2018, In : Journal of Clinical Investigation. 128, 10, p. 4604-4621 18 p.

Research output: Contribution to journalArticle

Open Access
Intermediate Filaments
Graft vs Host Disease
Vimentin
Regulatory T-Lymphocytes
Protein Kinase C
2017
14 Citations (Scopus)

Angiotensin-converting enzyme 2 amplification limited to the circulation does not protect mice from development of diabetic nephropathy

Wysocki, J., Ye, M., Khattab, A. M., Fogo, A., Martin, A., David, N. V., Kanwar, Y., Osborn, M. & Batlle, D., Jun 2017, In : Kidney international. 91, 6, p. 1336-1346 11 p.

Research output: Contribution to journalArticle

Open Access
Diabetic Nephropathies
Albuminuria
Renin-Angiotensin System
Streptozocin
angiotensin converting enzyme 2
4 Citations (Scopus)
Open Access
Iduronidase
Mucopolysaccharidosis I
Angiotensin Receptors
Renin-Angiotensin System
Pathology
10 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 journalArticle

Clustered Regularly Interspaced Short Palindromic Repeats
anemias
Fanconi Anemia
genes
Genes
13 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 journalArticle

Open Access
Clustered Regularly Interspaced Short Palindromic Repeats
T-Cell Antigen Receptor
Genome
Guide RNA
Integrases
5 Citations (Scopus)

Granulocyte colony-stimulating factor (GCSF) fused with Fc Domain produced from E. coli is less effective than Polyethylene Glycol-conjugated GCSF

Do, B. H., Kang, H. J., Song, J. A., Nguyen, M. T., Park, S., Yoo, J., Nguyen, A. N., Kwon, G. G., Jang, J., Jang, M., Lee, S., So, S., Sim, S., Lee, K. J., Osborn, M. J. & Choe, H., Dec 1 2017, In : Scientific reports. 7, 1, 6480.

Research output: Contribution to journalArticle

Open Access
Granulocyte Colony-Stimulating Factor
Escherichia coli
Proteins
Maltose-Binding Proteins
Myeloid Leukemia
8 Citations (Scopus)

Prokaryotic soluble expression and purification of bioactive human fibroblast growth factor 21 using maltose-binding protein

Nguyen, A. N., Song, J. A., Nguyen, M. T., Do, B. H., Kwon, G. G., Park, S. S., Yoo, J., Jang, J., Jin, J., Osborn, M. J., Jang, Y. J., Thi Vu, T. T., Oh, H. B. & Choe, H., Dec 1 2017, In : Scientific reports. 7, 1, 16139.

Research output: Contribution to journalArticle

Open Access
Maltose-Binding Proteins
His-His-His-His-His-His
Solubility
Protein Disulfide-Isomerases
Proteins
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 journalArticle

Open Access
Epidermolysis Bullosa Dystrophica
Immunosuppressive Agents
Mesenchymal Stromal Cells
Skin
Clustered Regularly Interspaced Short Palindromic Repeats
3 Citations (Scopus)

Soluble expression and purification of bioactive interleukin 33 in E. coli

Do, B. H., Park, S., Kwon, G. G., Nguyen, M. T., Kang, H. J., Song, J. A., Yoo, J., Nguyen, A. N., Jang, J., Jang, M., Lee, S., So, S., Sim, S., Jin, J., Lee, K. J., Osborn, M. J. & Choe, H., Jun 1 2017, In : Biotechnology and Bioprocess Engineering. 22, 3, p. 256-264 9 p.

Research output: Contribution to journalArticle

Maltose
Escherichia coli
Purification
Proteins
Affinity chromatography
2016
14 Citations (Scopus)
Clustered Regularly Interspaced Short Palindromic Repeats
Cell Engineering
Fanconi Anemia
Induced Pluripotent Stem Cells
Genes
67 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 journalArticle

Clustered Regularly Interspaced Short Palindromic Repeats
T-Lymphocytes
T-Cell Antigen Receptor
Adoptive Immunotherapy
Neoplasm Antigens
2015
28 Citations (Scopus)

B7-H3 expression in donor T cells and host cells negatively regulates acute graft-versus-host disease lethality

Veenstra, R. G., Flynn, R., Kreymborg, K., McDonald-Hyman, C., Saha, A., Taylor, P. A., Osborn, M. J., Panoskaltsis-Mortari, A., Schmitt-Graeff, A., Lieberknect, E., Murphy, W. J., Serody, J. S., Munn, D. H., Freeman, G. J., Allison, J. P., Mak, T. W., van den Brink, M., Zeiser, R. & Blazar, B. R., Jan 1 2015, In : Blood. 125, 21, p. 3335-3346 12 p.

Research output: Contribution to journalArticle

T-cells
Graft vs Host Disease
Grafts
T-Lymphocytes
Transplants
70 Citations (Scopus)

Fanconi anemia gene editing by the CRISPR/Cas9 system

Osborn, M. J., Gabriel, R., Webber, B. R., Defeo, A. P., McElroy, A. N., Jarjour, J., Starker, C. G., Wagner, J. E., Joung, J. K., Voytas, D. F., Von Kalle, C., Schmidt, M., Blazar, B. R. & Tolar, J., Feb 1 2015, In : Human gene therapy. 26, 2, p. 114-126 13 p.

Research output: Contribution to journalArticle

Clustered Regularly Interspaced Short Palindromic Repeats
Fanconi Anemia
Deoxyribonuclease I
DNA Repair-Deficiency Disorders
Genome
2014
48 Citations (Scopus)
Epidermolysis Bullosa Dystrophica
Gene therapy
Induced Pluripotent Stem Cells
Stem cells
Genes
2013
37 Citations (Scopus)

Minicircle DNA is superior to plasmid DNA in eliciting antigen-specific CD8 + T-cell responses

Dietz, W. M., Skinner, N. E. B., Hamilton, S. E., Jund, M. D., Heitfeld, S. M., Litterman, A. J., Hwu, P., Chen, Z. Y., Salazar, A. M., Ohlfest, J. R., Blazar, B. R., Pennell, C. A. & Osborn, M. J., Aug 2013, In : Molecular Therapy. 21, 8, p. 1526-1535 10 p.

Research output: Contribution to journalArticle

CD8 Antigens
Plasmids
T-Lymphocytes
DNA
Transgenes
166 Citations (Scopus)

TALEN-based gene correction for epidermolysis bullosa

Osborn, M. J., Starker, C. G., McElroy, A. N., Webber, B. R., Riddle, M. J., Xia, L., Defeo, A. P., Gabriel, R., Schmidt, M., Von Kalle, C., Carlson, D. F., Maeder, M. L., Joung, J. K., Wagner, J. E., Voytas, D. F., Blazar, B. R. & Tolar, J., Jun 2013, In : Molecular Therapy. 21, 6, p. 1151-1159 9 p.

Research output: Contribution to journalArticle

Epidermolysis Bullosa
Collagen Type VII
Genes
Epidermolysis Bullosa Dystrophica
High-Throughput Nucleotide Sequencing
2012
74 Citations (Scopus)

Targeting G with TAL Effectors: A Comparison of Activities of TALENs Constructed with NN and NK Repeat Variable Di-Residues

Christian, M. L., Demorest, Z. L., Starker, C. G., Osborn, M. J., Nyquist, M. D., Zhang, Y., Carlson, D. F., Bradley, P., Bogdanove, A. J. & Voytas, D. F., Sep 24 2012, In : PloS one. 7, 9, e45383.

Research output: Contribution to journalArticle

nucleases
Transcription
transcription (genetics)
Amino Acids
Yeast
2011
63 Citations (Scopus)
Iduronidase
Mucopolysaccharidosis I
Induced Pluripotent Stem Cells
Stem cells
Grafts
109 Citations (Scopus)
Epidermolysis Bullosa Dystrophica
Induced Pluripotent Stem Cells
Stem cells
Skin
Erosion
68 Citations (Scopus)
Iduronidase
Mucopolysaccharidoses
Genetic Therapy
Mucopolysaccharidosis I
DNA
14 Citations (Scopus)

Synthetic zinc finger nuclease design and rapid assembly

Osborn, M. J., Defeo, A. P., Blazar, B. R. & Tolar, J., Sep 1 2011, In : Human gene therapy. 22, 9, p. 1155-1165 11 p.

Research output: Contribution to journalArticle

Zinc Fingers
Iduronidase
Oligonucleotides
Mucopolysaccharidosis I
Lysosomal Storage Diseases
2010
206 Citations (Scopus)

Bone marrow transplantation for recessive dystrophic epidermolysis bullosa

Wagner, J. E., Ishida-Yamamoto, A., McGrath, J. A., Hordinsky, M., Keene, D. R., Riddle, M. J., Osborn, M. J., Lund, T., Dolan, M., Blazar, B. R. & Tolar, J., Aug 12 2010, In : New England Journal of Medicine. 363, 7, p. 629-639 11 p.

Research output: Contribution to journalArticle

Epidermolysis Bullosa Dystrophica
Bone Marrow Transplantation
Skin
Transplantation
Blister
32 Citations (Scopus)

Short-term inhibition of p53 combined with keratinocyte growth factor improves thymic epithelial cell recovery and enhances T-cell reconstitution after murine bone marrow transplantation

Kelly, R. M., Goren, E. M., Taylor, P. A., Mueller, S. N., Stefanski, H. E., Osborn, M. J., Scott, H. S., Komarova, E. A., Gudkov, A. V., Holländer, G. A. & Blazar, B. R., Feb 4 2010, In : Blood. 115, 5, p. 1088-1097 10 p.

Research output: Contribution to journalArticle

Fibroblast Growth Factor 7
T-cells
Bone Marrow Transplantation
Bone
Epithelial Cells
2009
119 Citations (Scopus)

Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells

Tolar, J., Akemi, I. Y., Riddle, M., McElmurry, R. T., Osborn, M., Xia, L., Lund, T., Slattery, C., Uitto, J., Christiano, A. M., Wagner, J. E. & Blazar, B. R., Jan 29 2009, In : Blood. 113, 5, p. 1167-1174 8 p.

Research output: Contribution to journalArticle

Collagen Type VII
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa
Bone Marrow Cells
Bone
2008
33 Citations (Scopus)

Targeting of the CNS in MPS-IH using a nonviral transferrin-α-L-iduronidase fusion gene product

Osborn, M. J., McElmurry, R. T., Peacock, B., Tolar, J. & Blazar, B. R., Aug 1 2008, In : Molecular Therapy. 16, 8, p. 1459-1466 8 p.

Research output: Contribution to journalArticle

Iduronidase
Gene Fusion
Transferrin
Mucopolysaccharidosis I
Central Nervous System
2007
494 Citations (Scopus)

Sarcoma derived from cultured mesenchymal stem cells

Tolar, J., Nauta, A. J., Osborn, M. J., Mortari, A. P., McElmurry, R. T., Bell, S., Xia, L., Zhou, N., Riddle, M., Schroeder, T. M., Westendorf, J. J., McIvor, R. S., Hogendoorn, P. C. W., Szuhai, K., Oseth, L., Hirsch, B., Yant, S. R., Kay, M. A., Peister, A., Prockop, D. J. & 2 others, Fibbe, W. E. & Blazar, B. R., Feb 1 2007, In : STEM CELLS. 25, 2, p. 371-379 9 p.

Research output: Contribution to journalArticle

Mesenchymal Stromal Cells
Sarcoma
Luciferases
Chromosome Aberrations
Bone Marrow
2005
53 Citations (Scopus)
Iduronidase
Picornaviridae
Firefly Luciferases
Gene Expression
Luciferases
32 Citations (Scopus)

Real-time in vivo imaging of stem cells following transgenesis by transposition

Tolar, J., Osborn, M., Bell, S., McElmurry, R., Xia, L., Riddle, M., Panoskaltsis-Mortari, A., Jiang, Y., McIvor, R. S., Contag, C. H., Yant, S. R., Kay, M. A., Verfaillie, C. M. & Blazar, B. R., Jul 1 2005, In : Molecular Therapy. 12, 1, p. 42-48 7 p.

Research output: Contribution to journalArticle

Gene Transfer Techniques
Stem Cells
Beauty
Clone Cells
Firefly Luciferases
2004
35 Citations (Scopus)

Overexpression of murine TSLP impairs lymphopoiesis and myelopoiesis

Osborn, M. J., Ryan, P. L., Kirchhof, N., Panoskaltsis-Mortari, A., Mortari, F. & Tudor, K. S. R. S., Feb 1 2004, In : Blood. 103, 3, p. 843-851 9 p.

Research output: Contribution to journalArticle

Lymphopoiesis
Myelopoiesis
Lymphocytes
Macrophages
Stromal Cells