Amalfitano A - Search Results

1

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

Sun B, Zhang H, Franco LM, Young SP, Schneider A, Bird A, Amalfitano A, Chen YT, Koeberl DD. Sun B, et al. Among authors: amalfitano a. Mol Ther. 2005 Jan;11(1):57-65. doi: 10.1016/j.ymthe.2004.10.004. Mol Ther. 2005. PMID: 15585406 Free article.

2

Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.

Sun BD, Chen YT, Bird A, Amalfitano A, Koeberl DD. Sun BD, et al. Among authors: amalfitano a. Mol Ther. 2003 Feb;7(2):193-201. doi: 10.1016/s1525-0016(02)00055-2. Mol Ther. 2003. PMID: 12597907 Free article.

3

Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector.

Sun B, Chen YT, Bird A, Xu F, Hou YX, Amalfitano A, Koeberl DD. Sun B, et al. Among authors: amalfitano a. Mol Ther. 2003 Apr;7(4):467-77. doi: 10.1016/s1525-0016(03)00022-4. Mol Ther. 2003. PMID: 12727109 Free article.

4

Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA.

Xu F, Ding E, Migone F, Serra D, Schneider A, Chen YT, Amalfitano A. Xu F, et al. Among authors: amalfitano a. J Gene Med. 2005 Feb;7(2):171-8. doi: 10.1002/jgm.660. J Gene Med. 2005. PMID: 15515143

5

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

Franco LM, Sun B, Yang X, Bird A, Zhang H, Schneider A, Brown T, Young SP, Clay TM, Amalfitano A, Chen YT, Koeberl DD. Franco LM, et al. Among authors: amalfitano a. Mol Ther. 2005 Nov;12(5):876-84. doi: 10.1016/j.ymthe.2005.04.024. Epub 2005 Jul 6. Mol Ther. 2005. PMID: 16005263 Free article.

6

Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction.

Ding E, Hu H, Hodges BL, Migone F, Serra D, Xu F, Chen YT, Amalfitano A. Ding E, et al. Among authors: amalfitano a. Mol Ther. 2002 Apr;5(4):436-46. doi: 10.1006/mthe.2002.0563. Mol Ther. 2002. PMID: 11945071 Free article.

7

Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease.

An Y, Young SP, Kishnani PS, Millington DS, Amalfitano A, Corz D, Chen YT. An Y, et al. Among authors: amalfitano a. Mol Genet Metab. 2005 Aug;85(4):247-54. doi: 10.1016/j.ymgme.2005.03.010. Mol Genet Metab. 2005. PMID: 15886040

8

Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice.

Kiang A, Hartman ZC, Liao S, Xu F, Serra D, Palmer DJ, Ng P, Amalfitano A. Kiang A, et al. Among authors: amalfitano a. Mol Ther. 2006 Jan;13(1):127-34. doi: 10.1016/j.ymthe.2005.08.006. Epub 2005 Oct 5. Mol Ther. 2006. PMID: 16169280 Free article.

9

Flupirtine blocks apoptosis in batten patient lymphoblasts and in human postmitotic CLN3- and CLN2-deficient neurons.

Dhar S, Bitting RL, Rylova SN, Jansen PJ, Lockhart E, Koeberl DD, Amalfitano A, Boustany RM. Dhar S, et al. Among authors: amalfitano a. Ann Neurol. 2002 Apr;51(4):448-66. doi: 10.1002/ana.10143. Ann Neurol. 2002. PMID: 11921051

10

Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells.

Banugaria SG, Patel TT, Mackey J, Das S, Amalfitano A, Rosenberg AS, Charrow J, Chen YT, Kishnani PS. Banugaria SG, et al. Among authors: amalfitano a. Mol Genet Metab. 2012 Apr;105(4):677-80. doi: 10.1016/j.ymgme.2012.01.019. Epub 2012 Jan 28. Mol Genet Metab. 2012. PMID: 22365055 Free PMC article.

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