Amalfitano A - Search Results

1

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.

Amalfitano A, Bengur AR, Morse RP, Majure JM, Case LE, Veerling DL, Mackey J, Kishnani P, Smith W, McVie-Wylie A, Sullivan JA, Hoganson GE, Phillips JA 3rd, Schaefer GB, Charrow J, Ware RE, Bossen EH, Chen YT. Amalfitano A, et al. Genet Med. 2001 Mar-Apr;3(2):132-8. Genet Med. 2001. PMID: 11286229 Free article. Clinical Trial.

2

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase.

Amalfitano A, McVie-Wylie AJ, Hu H, Dawson TL, Raben N, Plotz P, Chen YT. Amalfitano A, et al. Proc Natl Acad Sci U S A. 1999 Aug 3;96(16):8861-6. doi: 10.1073/pnas.96.16.8861. Proc Natl Acad Sci U S A. 1999. PMID: 10430861 Free PMC article.

3

Towards a molecular therapy for glycogen storage disease type II (Pompe disease).

Chen YT, Amalfitano A. Chen YT, et al. Among authors: amalfitano a. Mol Med Today. 2000 Jun;6(6):245-51. doi: 10.1016/s1357-4310(00)01694-4. Mol Med Today. 2000. PMID: 10840383 Review.

4

Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice.

Ding EY, Hodges BL, Hu H, McVie-Wylie AJ, Serra D, Migone FK, Pressley D, Chen YT, Amalfitano A. Ding EY, et al. Among authors: amalfitano a. Hum Gene Ther. 2001 May 20;12(8):955-65. doi: 10.1089/104303401750195917. Hum Gene Ther. 2001. PMID: 11387060

5

Multiple muscles in the AMD quail can be "cross-corrected" of pathologic glycogen accumulation after intravenous injection of an [E1-, polymerase-] adenovirus vector encoding human acid-alpha-glucosidase.

McVie-Wylie AJ, Ding EY, Lawson T, Serra D, Migone FK, Pressley D, Mizutani M, Kikuchi T, Chen YT, Amalfitano A. McVie-Wylie AJ, et al. Among authors: amalfitano a. J Gene Med. 2003 May;5(5):399-406. doi: 10.1002/jgm.355. J Gene Med. 2003. PMID: 12731088

6

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.

Kishnani PS, Nicolino M, Voit T, Rogers RC, Tsai AC, Waterson J, Herman GE, Amalfitano A, Thurberg BL, Richards S, Davison M, Corzo D, Chen YT. Kishnani PS, et al. Among authors: amalfitano a. J Pediatr. 2006 Jul;149(1):89-97. doi: 10.1016/j.jpeds.2006.02.035. J Pediatr. 2006. PMID: 16860134 Free PMC article. Clinical Trial.

7

Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.

Xu F, Ding E, Liao SX, Migone F, Dai J, Schneider A, Serra D, Chen YT, Amalfitano A. Xu F, et al. Among authors: amalfitano a. Gene Ther. 2004 Nov;11(21):1590-8. doi: 10.1038/sj.gt.3302314. Gene Ther. 2004. PMID: 15356673

8

Dubowitz syndrome: a defect in the cholesterol biosynthetic pathway?

Ahmad A, Amalfitano A, Chen YT, Kishnani PS, Miller C, Kelley R. Ahmad A, et al. Among authors: amalfitano a. Am J Med Genet. 1999 Oct 29;86(5):503-4. Am J Med Genet. 1999. PMID: 10508998 No abstract available.

9

Long-term, high-level hepatic secretion of acid α-glucosidase for Pompe disease achieved in non-human primates using helper-dependent adenovirus.

Rastall DP, Seregin SS, Aldhamen YA, Kaiser LM, Mullins C, Liou A, Ing F, Pereria-Hicks C, Godbehere-Roosa S, Palmer D, Ng P, Amalfitano A. Rastall DP, et al. Among authors: amalfitano a. Gene Ther. 2016 Oct;23(10):743-752. doi: 10.1038/gt.2016.53. Epub 2016 Jul 1. Gene Ther. 2016. PMID: 27367841

10

Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy.

Ahmad A, Brinson M, Hodges BL, Chamberlain JS, Amalfitano A. Ahmad A, et al. Among authors: amalfitano a. Hum Mol Genet. 2000 Oct 12;9(17):2507-15. doi: 10.1093/hmg/9.17.2507. Hum Mol Genet. 2000. PMID: 11030755

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