Osborn MJ - Search Results

1

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

Kim YM, Yum MS, Heo SH, Kim T, Jin HK, Bae JS, Seo GH, Oh A, Yoon HM, Lim HT, Kim HW, Ko TS, Lim HS, Osborn MJ, Tolar J, Cozma C, Rolfs A, Zimran A, Lee BH, Yoo HW. Kim YM, et al. Among authors: osborn mj. J Med Genet. 2020 Feb;57(2):124-131. doi: 10.1136/jmedgenet-2019-106132. Epub 2019 Oct 24. J Med Genet. 2020. PMID: 31649052 Free PMC article.

2

A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system.

Osborn MJ, Panoskaltsis-Mortari A, McElmurry RT, Bell SK, Vignali DA, Ryan MD, Wilber AC, McIvor RS, Tolar J, Blazar BR. Osborn MJ, et al. Mol Ther. 2005 Sep;12(3):569-74. doi: 10.1016/j.ymthe.2005.04.013. Mol Ther. 2005. PMID: 15964244 Free article.

3

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

Osborn MJ, McElmurry RT, Peacock B, Tolar J, Blazar BR. Osborn MJ, et al. Mol Ther. 2008 Aug;16(8):1459-66. doi: 10.1038/mt.2008.119. Epub 2008 Jun 3. Mol Ther. 2008. PMID: 18523448 Free PMC article.

4

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

Tolar J, Ishida-Yamamoto A, Riddle M, McElmurry RT, Osborn M, Xia L, Lund T, Slattery C, Uitto J, Christiano AM, Wagner JE, Blazar BR. Tolar J, et al. Blood. 2009 Jan 29;113(5):1167-74. doi: 10.1182/blood-2008-06-161299. Epub 2008 Oct 27. Blood. 2009. PMID: 18955559 Free PMC article.

5

Bone marrow transplantation for recessive dystrophic epidermolysis bullosa.

Wagner JE, Ishida-Yamamoto A, McGrath JA, Hordinsky M, Keene DR, Woodley DT, Chen M, Riddle MJ, Osborn MJ, Lund T, Dolan M, Blazar BR, Tolar J. Wagner JE, et al. Among authors: osborn mj. N Engl J Med. 2010 Aug 12;363(7):629-39. doi: 10.1056/NEJMoa0910501. N Engl J Med. 2010. PMID: 20818854 Free PMC article. Clinical Trial.

6

Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I.

Osborn MJ, McElmurry RT, Lees CJ, DeFeo AP, Chen ZY, Kay MA, Naldini L, Freeman G, Tolar J, Blazar BR. Osborn MJ, et al. Mol Ther. 2011 Mar;19(3):450-60. doi: 10.1038/mt.2010.249. Epub 2010 Nov 16. Mol Ther. 2011. PMID: 21081900 Free PMC article.

7

Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa.

Tolar J, Xia L, Riddle MJ, Lees CJ, Eide CR, McElmurry RT, Titeux M, Osborn MJ, Lund TC, Hovnanian A, Wagner JE, Blazar BR. Tolar J, et al. Among authors: osborn mj. J Invest Dermatol. 2011 Apr;131(4):848-56. doi: 10.1038/jid.2010.346. Epub 2010 Dec 2. J Invest Dermatol. 2011. PMID: 21124339 Free PMC article.

8

Synthetic zinc finger nuclease design and rapid assembly.

Osborn MJ, DeFeo AP, Blazar BR, Tolar J. Osborn MJ, et al. Hum Gene Ther. 2011 Sep;22(9):1155-65. doi: 10.1089/hum.2011.072. Epub 2011 Aug 10. Hum Gene Ther. 2011. PMID: 21663559 Free PMC article.

9

TALEN-based gene correction for epidermolysis bullosa.

Osborn MJ, Starker CG, McElroy AN, Webber BR, Riddle MJ, Xia L, DeFeo AP, Gabriel R, Schmidt M, von Kalle C, Carlson DF, Maeder ML, Joung JK, Wagner JE, Voytas DF, Blazar BR, Tolar J. Osborn MJ, et al. Mol Ther. 2013 Jun;21(6):1151-9. doi: 10.1038/mt.2013.56. Epub 2013 Apr 2. Mol Ther. 2013. PMID: 23546300 Free PMC article.

10

Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa.

Perdoni C, Osborn MJ, Tolar J. Perdoni C, et al. Among authors: osborn mj. Transl Res. 2016 Feb;168:50-58. doi: 10.1016/j.trsl.2015.05.008. Epub 2015 May 27. Transl Res. 2016. PMID: 26073463 Free PMC article. Review.

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