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Items: 13

1.

Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG.

Iyer S, Sam FS, DiPrimio N, Preston G, Verheijen J, Murthy K, Parton Z, Tsang H, Lao J, Morava E, Perlstein EO.

Dis Model Mech. 2019 Nov 11;12(11). pii: dmm040584. doi: 10.1242/dmm.040584.

2.

Drug screens of NGLY1 deficiency in worm and fly models reveal catecholamine, NRF2 and anti-inflammatory-pathway activation as potential clinical approaches.

Iyer S, Mast JD, Tsang H, Rodriguez TP, DiPrimio N, Prangley M, Sam FS, Parton Z, Perlstein EO.

Dis Model Mech. 2019 Nov 4;12(11). pii: dmm040576. doi: 10.1242/dmm.040576.

3.

Yeast Models of Phosphomannomutase 2 Deficiency, a Congenital Disorder of Glycosylation.

Lao JP, DiPrimio N, Prangley M, Sam FS, Mast JD, Perlstein EO.

G3 (Bethesda). 2019 Feb 7;9(2):413-423. doi: 10.1534/g3.118.200934.

4.

GEX1A, a Polyketide from Streptomyces chromofuscus, Corrects the Cellular Defects Associated with Niemann-Pick Type C1 in Human Fibroblasts.

Granatosky EA, DiPrimio N, Pickering JRE, Stevens DC, Perlstein EO, Taylor RE.

J Nat Prod. 2018 Sep 28;81(9):2018-2025. doi: 10.1021/acs.jnatprod.8b00314. Epub 2018 Sep 6.

PMID:
30188717
5.

The designability of protein switches by chemical rescue of structure: mechanisms of inactivation and reactivation.

Xia Y, DiPrimio N, Keppel TR, Vo B, Fraser K, Battaile KP, Egan C, Bystroff C, Lovell S, Weis DD, Anderson JC, Karanicolas J.

J Am Chem Soc. 2013 Dec 18;135(50):18840-9. doi: 10.1021/ja407644b. Epub 2013 Dec 6.

6.

Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles.

Li C, Diprimio N, Bowles DE, Hirsch ML, Monahan PE, Asokan A, Rabinowitz J, Agbandje-McKenna M, Samulski RJ.

J Virol. 2012 Aug;86(15):7752-9. doi: 10.1128/JVI.00675-12. Epub 2012 May 16.

7.

Adeno-associated virus for the treatment of muscle diseases: toward clinical trials.

DiPrimio N, McPhee SW, Samulski RJ.

Curr Opin Mol Ther. 2010 Oct;12(5):553-60. Review.

PMID:
20886387
8.

Mutagenesis of adeno-associated virus type 2 capsid protein VP1 uncovers new roles for basic amino acids in trafficking and cell-specific transduction.

Johnson JS, Li C, DiPrimio N, Weinberg MS, McCown TJ, Samulski RJ.

J Virol. 2010 Sep;84(17):8888-902. doi: 10.1128/JVI.00687-10. Epub 2010 Jun 23.

9.

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle.

Asokan A, Conway JC, Phillips JL, Li C, Hegge J, Sinnott R, Yadav S, DiPrimio N, Nam HJ, Agbandje-McKenna M, McPhee S, Wolff J, Samulski RJ.

Nat Biotechnol. 2010 Jan;28(1):79-82. doi: 10.1038/nbt.1599. Epub 2009 Dec 27.

10.

Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo.

Li C, Hirsch M, DiPrimio N, Asokan A, Goudy K, Tisch R, Samulski RJ.

J Virol. 2009 Jul;83(13):6817-24. doi: 10.1128/JVI.00278-09. Epub 2009 Apr 15.

11.

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles.

Li W, Asokan A, Wu Z, Van Dyke T, DiPrimio N, Johnson JS, Govindaswamy L, Agbandje-McKenna M, Leichtle S, Eugene Redmond D Jr, McCown TJ, Petermann KB, Sharpless NE, Samulski RJ.

Mol Ther. 2008 Jul;16(7):1252-1260. doi: 10.1038/mt.2008.100. Epub 2016 Dec 8.

PMID:
28178482
12.

Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles.

Li W, Asokan A, Wu Z, Van Dyke T, DiPrimio N, Johnson JS, Govindaswamy L, Agbandje-McKenna M, Leichtle S, Redmond DE Jr, McCown TJ, Petermann KB, Sharpless NE, Samulski RJ.

Mol Ther. 2008 Jul;16(7):1252-60. doi: 10.1038/mt.2008.100. Epub 2008 May 20.

13.

Surface loop dynamics in adeno-associated virus capsid assembly.

DiPrimio N, Asokan A, Govindasamy L, Agbandje-McKenna M, Samulski RJ.

J Virol. 2008 Jun;82(11):5178-89. doi: 10.1128/JVI.02721-07. Epub 2008 Mar 26.

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