Ziegler R - Search Results

1

Reversibility of neuropathology in Tay-Sachs-related diseases.

Cachón-González MB, Wang SZ, Ziegler R, Cheng SH, Cox TM. Cachón-González MB, et al. Among authors: ziegler r. Hum Mol Genet. 2014 Feb 1;23(3):730-48. doi: 10.1093/hmg/ddt459. Epub 2013 Sep 20. Hum Mol Genet. 2014. PMID: 24057669 Free PMC article.

2

Effective gene therapy in an authentic model of Tay-Sachs-related diseases.

Cachón-González MB, Wang SZ, Lynch A, Ziegler R, Cheng SH, Cox TM. Cachón-González MB, et al. Among authors: ziegler r. Proc Natl Acad Sci U S A. 2006 Jul 5;103(27):10373-10378. doi: 10.1073/pnas.0603765103. Epub 2006 Jun 26. Proc Natl Acad Sci U S A. 2006. PMID: 16801539 Free PMC article.

3

Gene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flow.

Cachón-González MB, Wang SZ, McNair R, Bradley J, Lunn D, Ziegler R, Cheng SH, Cox TM. Cachón-González MB, et al. Among authors: ziegler r. Mol Ther. 2012 Aug;20(8):1489-500. doi: 10.1038/mt.2012.44. Epub 2012 Mar 27. Mol Ther. 2012. PMID: 22453766 Free PMC article.

4

Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.

Sargeant TJ, Wang S, Bradley J, Smith NJ, Raha AA, McNair R, Ziegler RJ, Cheng SH, Cox TM, Cachón-González MB. Sargeant TJ, et al. Among authors: ziegler rj. Hum Mol Genet. 2011 Nov 15;20(22):4371-80. doi: 10.1093/hmg/ddr364. Epub 2011 Aug 18. Hum Mol Genet. 2011. PMID: 21852247

5

Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.

Ziegler RJ, Bercury SD, Fidler J, Zhao MA, Foley J, Taksir TV, Ryan S, Hodges BL, Scheule RK, Shihabuddin LS, Cheng SH. Ziegler RJ, et al. Hum Gene Ther. 2008 Jun;19(6):609-21. doi: 10.1089/hum.2008.010. Hum Gene Ther. 2008. PMID: 18500944

6

Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease.

Ziegler RJ, Brown C, Barbon CM, D'Angona AM, Schuchman EH, Andrews L, Thurberg BL, McPherson JM, Karey KP, Cheng SH. Ziegler RJ, et al. Mol Genet Metab. 2009 May;97(1):35-42. doi: 10.1016/j.ymgme.2009.01.008. Epub 2009 Jan 25. Mol Genet Metab. 2009. PMID: 19231265

7

Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease.

Ziegler RJ, Li C, Cherry M, Zhu Y, Hempel D, van Rooijen N, Ioannou YA, Desnick RJ, Goldberg MA, Yew NS, Cheng SH. Ziegler RJ, et al. Hum Gene Ther. 2002 May 20;13(8):935-45. doi: 10.1089/10430340252939041. Hum Gene Ther. 2002. PMID: 12031126

8

AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease.

Barbon CM, Ziegler RJ, Li C, Armentano D, Cherry M, Desnick RJ, Schuchman EH, Cheng SH. Barbon CM, et al. Mol Ther. 2005 Sep;12(3):431-40. doi: 10.1016/j.ymthe.2005.03.011. Mol Ther. 2005. PMID: 16099409 Free article.

9

Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A.

Ziegler RJ, Cherry M, Barbon CM, Li C, Bercury SD, Armentano D, Desnick RJ, Cheng SH. Ziegler RJ, et al. Mol Ther. 2007 Mar;15(3):492-500. doi: 10.1038/sj.mt.6300066. Epub 2006 Dec 26. Mol Ther. 2007. PMID: 17191071 Free article.

10

Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice.

Foley JW, Bercury SD, Finn P, Cheng SH, Scheule RK, Ziegler RJ. Foley JW, et al. Mol Ther. 2010 Sep;18(9):1584-91. doi: 10.1038/mt.2010.110. Epub 2010 Jun 15. Mol Ther. 2010. PMID: 20551907 Free PMC article.

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