Muenzer J - Search Results

1

Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain.

Fu H, Muenzer J, Samulski RJ, Breese G, Sifford J, Zeng X, McCarty DM. Fu H, et al. Among authors: muenzer j. Mol Ther. 2003 Dec;8(6):911-7. doi: 10.1016/j.ymthe.2003.08.021. Mol Ther. 2003. PMID: 14664793 Free article.

2

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery.

Fu H, Samulski RJ, McCown TJ, Picornell YJ, Fletcher D, Muenzer J. Fu H, et al. Among authors: muenzer j. Mol Ther. 2002 Jan;5(1):42-9. doi: 10.1006/mthe.2001.0514. Mol Ther. 2002. PMID: 11786044 Free article.

3

Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery.

Fu H, DiRosario J, Kang L, Muenzer J, McCarty DM. Fu H, et al. Among authors: muenzer j. J Gene Med. 2010 Jul;12(7):624-33. doi: 10.1002/jgm.1480. J Gene Med. 2010. PMID: 20603889

4

Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.

Fan Z, Kocis K, Valley R, Howard JF, Chopra M, An H, Lin W, Muenzer J, Powers W. Fan Z, et al. Among authors: muenzer j. Mol Ther. 2012 Feb;20(2):456-61. doi: 10.1038/mt.2011.137. Epub 2011 Jul 19. Mol Ther. 2012. PMID: 21772257 Free PMC article.

5

Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice.

Fu H, Zaraspe K, Murakami N, Meadows AS, Pineda RJ, McCarty DM, Muenzer J. Fu H, et al. Among authors: muenzer j. Mol Ther Methods Clin Dev. 2018 Sep 4;10:327-340. doi: 10.1016/j.omtm.2018.07.005. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30191159 Free PMC article.

6

First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.

Harmatz P, Prada CE, Burton BK, Lau H, Kessler CM, Cao L, Falaleeva M, Villegas AG, Zeitler J, Meyer K, Miller W, Wong Po Foo C, Vaidya S, Swenson W, Shiue LH, Rouy D, Muenzer J. Harmatz P, et al. Among authors: muenzer j. Mol Ther. 2022 Dec 7;30(12):3587-3600. doi: 10.1016/j.ymthe.2022.10.010. Epub 2022 Oct 25. Mol Ther. 2022. PMID: 36299240 Free PMC article.

7

High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.

Fan Z, Kocis K, Valley R, Howard JF Jr, Chopra M, Chen Y, An H, Lin W, Muenzer J, Powers W. Fan Z, et al. Among authors: muenzer j. Hum Gene Ther. 2015 Sep;26(9):614-21. doi: 10.1089/hum.2015.023. Epub 2015 Jul 30. Hum Gene Ther. 2015. PMID: 25953425 Free PMC article.

8

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome).

Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A. Muenzer J, et al. Genet Med. 2006 Aug;8(8):465-73. doi: 10.1097/01.gim.0000232477.37660.fb. Genet Med. 2006. PMID: 16912578 Free article. Clinical Trial.

9

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome.

Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, Ramaswami U, Vellodi A, Wraith JE, Cleary M, Gucsavas-Calikoglu M, Puga AC, Shinawi M, Ulbrich B, Vijayaraghavan S, Wendt S, Conway AM, Rossi A, Whiteman DA, Kimura A. Muenzer J, et al. Genet Med. 2011 Feb;13(2):95-101. doi: 10.1097/GIM.0b013e3181fea459. Genet Med. 2011. PMID: 21150784 Free article. Clinical Trial.

10

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II.

Muenzer J, Hendriksz CJ, Fan Z, Vijayaraghavan S, Perry V, Santra S, Solanki GA, Mascelli MA, Pan L, Wang N, Sciarappa K, Barbier AJ. Muenzer J, et al. Genet Med. 2016 Jan;18(1):73-81. doi: 10.1038/gim.2015.36. Epub 2015 Apr 2. Genet Med. 2016. PMID: 25834948 Free article. Clinical Trial.

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