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Results: 11

Cited In for PubMed (Select 20163245)

1.

Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.

Han SO, Li S, Brooks ED, Masat E, Leborgne C, Banugaria S, Bird A, Mingozzi F, Waldmann H, Koeberl D.

Hum Gene Ther. 2015 Jan;26(1):26-35. doi: 10.1089/hum.2014.115.

PMID:
25382056
2.

Immunological ignorance allows long-term gene expression after perinatal recombinant adeno-associated virus-mediated gene transfer to murine airways.

Carlon MS, Vidović D, Dooley J, da Cunha MM, Maris M, Lampi Y, Toelen J, Van den Haute C, Baekelandt V, Deprest J, Verbeken E, Liston A, Gijsbers R, Debyser Z.

Hum Gene Ther. 2014 Jun;25(6):517-28. doi: 10.1089/hum.2013.196. Epub 2014 Mar 26.

3.

The liver as a target organ for gene therapy: state of the art, challenges, and future perspectives.

Jacobs F, Gordts SC, Muthuramu I, De Geest B.

Pharmaceuticals (Basel). 2012 Dec 10;5(12):1372-92. doi: 10.3390/ph5121372.

4.

Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine Glycogen Storage Disease type Ia.

Brooks ED, Little D, Arumugam R, Sun B, Curtis S, Demaster A, Maranzano M, Jackson MW, Kishnani P, Freemark MS, Koeberl DD.

Mol Genet Metab. 2013 Jun;109(2):161-70. doi: 10.1016/j.ymgme.2013.03.018. Epub 2013 Apr 6.

5.

Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia.

Demaster A, Luo X, Curtis S, Williams KD, Landau DJ, Drake EJ, Kozink DM, Bird A, Crane B, Sun F, Pinto CR, Brown TT, Kemper AR, Koeberl DD.

Hum Gene Ther. 2012 Apr;23(4):407-18. doi: 10.1089/hum.2011.106. Epub 2012 Mar 8.

6.

Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.

Wang L, Wang H, Bell P, McMenamin D, Wilson JM.

Hum Gene Ther. 2012 May;23(5):533-9. doi: 10.1089/hum.2011.183. Epub 2012 Feb 8.

7.

AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta).

Wang L, Bell P, Lin J, Calcedo R, Tarantal AF, Wilson JM.

Mol Ther. 2011 Nov;19(11):2012-20. doi: 10.1038/mt.2011.151. Epub 2011 Aug 2.

8.

Perinatal gene transfer to the liver.

McKay TR, Rahim AA, Buckley SM, Ward NJ, Chan JK, Howe SJ, Waddington SN.

Curr Pharm Des. 2011;17(24):2528-41. Review.

9.

Glucose-6-phosphatase deficiency.

Froissart R, Piraud M, Boudjemline AM, Vianey-Saban C, Petit F, Hubert-Buron A, Eberschweiler PT, Gajdos V, Labrune P.

Orphanet J Rare Dis. 2011 May 20;6:27. doi: 10.1186/1750-1172-6-27. Review.

10.

Recombinant AAV-directed gene therapy for type I glycogen storage diseases.

Chou JY, Mansfield BC.

Expert Opin Biol Ther. 2011 Aug;11(8):1011-24. doi: 10.1517/14712598.2011.578067. Epub 2011 Apr 20. Review.

11.

Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease.

Specht A, Fiske L, Erger K, Cossette T, Verstegen J, Campbell-Thompson M, Struck MB, Lee YM, Chou JY, Byrne BJ, Correia CE, Mah CS, Weinstein DA, Conlon TJ.

J Biomed Biotechnol. 2011;2011:646257. doi: 10.1155/2011/646257. Epub 2011 Jan 3. Review.

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