Raben N - Search Results

1

Nutritional co-therapy with 1,3-butanediol and multi-ingredient antioxidants enhances autophagic clearance in Pompe disease.

Nilsson MI, Crozier M, Di Carlo A, Xhuti D, Manta K, Roik LJ, Bujak AL, Nederveen JP, Tarnopolsky MG, Hettinga B, Meena NK, Raben N, Tarnopolsky MA. Nilsson MI, et al. Among authors: raben n. Mol Genet Metab. 2022 Sep-Oct;137(1-2):228-240. doi: 10.1016/j.ymgme.2022.06.001. Epub 2022 Jun 11. Mol Genet Metab. 2022. PMID: 35718712

2

Enzyme replacement therapy in the mouse model of Pompe disease.

Raben N, Danon M, Gilbert AL, Dwivedi S, Collins B, Thurberg BL, Mattaliano RJ, Nagaraju K, Plotz PH. Raben N, et al. Mol Genet Metab. 2003 Sep-Oct;80(1-2):159-69. doi: 10.1016/j.ymgme.2003.08.022. Mol Genet Metab. 2003. PMID: 14567965

3

Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease.

McVie-Wylie AJ, Lee KL, Qiu H, Jin X, Do H, Gotschall R, Thurberg BL, Rogers C, Raben N, O'Callaghan M, Canfield W, Andrews L, McPherson JM, Mattaliano RJ. McVie-Wylie AJ, et al. Among authors: raben n. Mol Genet Metab. 2008 Aug;94(4):448-455. doi: 10.1016/j.ymgme.2008.04.009. Epub 2008 Jun 5. Mol Genet Metab. 2008. PMID: 18538603 Free PMC article.

4

Murine muscle cell models for Pompe disease and their use in studying therapeutic approaches.

Takikita S, Myerowitz R, Zaal K, Raben N, Plotz PH. Takikita S, et al. Among authors: raben n. Mol Genet Metab. 2009 Apr;96(4):208-17. doi: 10.1016/j.ymgme.2008.12.012. Epub 2009 Jan 22. Mol Genet Metab. 2009. PMID: 19167256 Free PMC article.

To evaluate substrate deprivation as a strategy to prevent the accumulation of lysosomal glycogen, we knocked down Atg7, a gene essential for autophagosome formation, via siRNA, but we observed no effect on the extent of glycogen accumulation, thus confirming our recent observati …

To evaluate substrate deprivation as a strategy to prevent the accumulation of lysosomal glycogen, we knocked down Atg7, a gene essential fo …

5

Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy.

Raben N, Ralston E, Chien YH, Baum R, Schreiner C, Hwu WL, Zaal KJ, Plotz PH. Raben N, et al. Mol Genet Metab. 2010 Dec;101(4):324-31. doi: 10.1016/j.ymgme.2010.08.001. Epub 2010 Aug 7. Mol Genet Metab. 2010. PMID: 20801068 Free PMC article.

6

Pros and cons of different ways to address dysfunctional autophagy in Pompe disease.

Lim JA, Meena NK, Raben N. Lim JA, et al. Among authors: raben n. Ann Transl Med. 2019 Jul;7(13):279. doi: 10.21037/atm.2019.03.51. Ann Transl Med. 2019. PMID: 31392191 Free PMC article. Review.

7

Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease.

Meena NK, Ralston E, Raben N, Puertollano R. Meena NK, et al. Among authors: raben n. Mol Ther Methods Clin Dev. 2020 Jun 10;18:199-214. doi: 10.1016/j.omtm.2020.05.026. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32671132 Free PMC article.

8

Pompe Disease: New Developments in an Old Lysosomal Storage Disorder.

Meena NK, Raben N. Meena NK, et al. Among authors: raben n. Biomolecules. 2020 Sep 18;10(9):1339. doi: 10.3390/biom10091339. Biomolecules. 2020. PMID: 32962155 Free PMC article. Review.

9

Chemoenzymatic glycan-selective remodeling of a therapeutic lysosomal enzyme with high-affinity M6P-glycan ligands. Enzyme substrate specificity is the name of the game.

Zhang X, Liu H, Meena N, Li C, Zong G, Raben N, Puertollano R, Wang LX. Zhang X, et al. Among authors: raben n. Chem Sci. 2021 Aug 19;12(37):12451-12462. doi: 10.1039/d1sc03188k. eCollection 2021 Sep 29. Chem Sci. 2021. PMID: 34603676 Free PMC article.

10

AAV-mediated delivery of secreted acid α-glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice.

Meena NK, Randazzo D, Raben N, Puertollano R. Meena NK, et al. Among authors: raben n. JCI Insight. 2023 Aug 22;8(16):e170199. doi: 10.1172/jci.insight.170199. JCI Insight. 2023. PMID: 37463048 Free PMC article.

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