Desnick RJ - Search Results

1

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. Among authors: desnick rj. Mol Ther. 2005 Sep;12(3):431-40. doi: 10.1016/j.ymthe.2005.03.011. Mol Ther. 2005. PMID: 16099409 Free article.

2

Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.

Przybylska M, Wu IH, Zhao H, Ziegler RJ, Tousignant JD, Desnick RJ, Scheule RK, Cheng SH, Yew NS. Przybylska M, et al. Among authors: desnick rj. J Gene Med. 2004 Jan;6(1):85-92. doi: 10.1002/jgm.468. J Gene Med. 2004. PMID: 14716680

3

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

Ziegler RJ, Lonning SM, Armentano D, Li C, Souza DW, Cherry M, Ford C, Barbon CM, Desnick RJ, Gao G, Wilson JM, Peluso R, Godwin S, Carter BJ, Gregory RJ, Wadsworth SC, Cheng SH. Ziegler RJ, et al. Among authors: desnick rj. Mol Ther. 2004 Feb;9(2):231-40. doi: 10.1016/j.ymthe.2003.11.015. Mol Ther. 2004. PMID: 14759807 Free article.

4

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. Among authors: desnick rj. 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.

5

Types A and B Niemann-Pick disease.

Schuchman EH, Desnick RJ. Schuchman EH, et al. Among authors: desnick rj. Mol Genet Metab. 2017 Jan-Feb;120(1-2):27-33. doi: 10.1016/j.ymgme.2016.12.008. Epub 2016 Dec 16. Mol Genet Metab. 2017. PMID: 28164782 Free PMC article. Review.

6

Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8-mediated Hepatic Expression of α-galactosidase A.

Ziegler RJ, Cherry M, Barbon CM, Li C, Bercury SD, Armentano D, Desnick RJ, Cheng SH. Ziegler RJ, et al. Among authors: desnick rj. Mol Ther. 2007 Mar;15(3):492-500. doi: 10.1038/sj.mt.6300066. Epub 2016 Dec 8. Mol Ther. 2007. PMID: 28182896

7

Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease.

Li C, Ziegler RJ, Cherry M, Lukason M, Desnick RJ, Yew NS, Cheng SH. Li C, et al. Among authors: desnick rj. Mol Ther. 2002 Jun;5(6):745-54. doi: 10.1006/mthe.2002.0605. Mol Ther. 2002. PMID: 12027559 Free article.

8

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. Among authors: desnick rj. Hum Gene Ther. 2002 May 20;13(8):935-45. doi: 10.1089/10430340252939041. Hum Gene Ther. 2002. PMID: 12031126

9

Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer.

Ziegler RJ, Yew NS, Li C, Cherry M, Berthelette P, Romanczuk H, Ioannou YA, Zeidner KM, Desnick RJ, Cheng SH. Ziegler RJ, et al. Among authors: desnick rj. Hum Gene Ther. 1999 Jul 1;10(10):1667-82. doi: 10.1089/10430349950017671. Hum Gene Ther. 1999. PMID: 10428212

10

The demographics and distribution of type B Niemann-Pick disease: novel mutations lead to new genotype/phenotype correlations.

Simonaro CM, Desnick RJ, McGovern MM, Wasserstein MP, Schuchman EH. Simonaro CM, et al. Among authors: desnick rj. Am J Hum Genet. 2002 Dec;71(6):1413-9. doi: 10.1086/345074. Epub 2002 Oct 4. Am J Hum Genet. 2002. PMID: 12369017 Free PMC article.

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