32785089 21654821 30859111 21599942 25224826 31319709 22310927 25382056 33348688 26865405 21732191 24548076 22185325 35060713 25356975 33357766 29428299 31227835 36006546 21774770 24281341 21774772 31890731 25288127 34258141 22098408 28755200 3071417 - Search Results

Year	Number of Results
2011	10
2012	5
2013	2
2014	4
2015	5
2016	2
2017	2
2018	3
2019	7
2020	2
2021	3
2022	2
2023	0

1

The Evolution of Gene Therapy in the Treatment of Metabolic Liver Diseases.

Moscoso CG, Steer CJ. Moscoso CG, et al. Genes (Basel). 2020 Aug 10;11(8):915. doi: 10.3390/genes11080915. Genes (Basel). 2020. PMID: 32785089 Free PMC article. Review.

2

Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia.

Crane B, Luo X, Demaster A, Williams KD, Kozink DM, Zhang P, Brown TT, Pinto CR, Oka K, Sun F, Jackson MW, Chan L, Koeberl DD. Crane B, et al. Gene Ther. 2012 Apr;19(4):443-52. doi: 10.1038/gt.2011.86. Epub 2011 Jun 9. Gene Ther. 2012. PMID: 21654821

3

Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.

Kang HR, Waskowicz L, Seifts AM, Landau DJ, Young SP, Koeberl DD. Kang HR, et al. Mol Ther Methods Clin Dev. 2019 Feb 10;13:265-273. doi: 10.1016/j.omtm.2019.02.002. eCollection 2019 Jun 14. Mol Ther Methods Clin Dev. 2019. PMID: 30859111 Free PMC article.

4

Glucose-6-phosphatase deficiency.

Froissart R, Piraud M, Boudjemline AM, Vianey-Saban C, Petit F, Hubert-Buron A, Eberschweiler PT, Gajdos V, Labrune P. Froissart R, et al. Orphanet J Rare Dis. 2011 May 20;6:27. doi: 10.1186/1750-1172-6-27. Orphanet J Rare Dis. 2011. PMID: 21599942 Free PMC article. Review.

5

Large animal models and new therapies for glycogen storage disease.

Brooks ED, Koeberl DD. Brooks ED, et al. J Inherit Metab Dis. 2015 May;38(3):505-9. doi: 10.1007/s10545-014-9766-8. Epub 2014 Sep 16. J Inherit Metab Dis. 2015. PMID: 25224826 Free PMC article. Review.

6

Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III.

Jauze L, Monteillet L, Mithieux G, Rajas F, Ronzitti G. Jauze L, et al. Hum Gene Ther. 2019 Oct;30(10):1263-1273. doi: 10.1089/hum.2019.102. Epub 2019 Aug 27. Hum Gene Ther. 2019. PMID: 31319709 Review.

7

In search of proof-of-concept: gene therapy for glycogen storage disease type Ia.

Koeberl DD. Koeberl DD. J Inherit Metab Dis. 2012 Jul;35(4):671-8. doi: 10.1007/s10545-012-9454-5. Epub 2012 Feb 7. J Inherit Metab Dis. 2012. PMID: 22310927

8

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. Han SO, et al. Hum Gene Ther. 2015 Jan;26(1):26-35. doi: 10.1089/hum.2014.115. Hum Gene Ther. 2015. PMID: 25382056 Free PMC article.

9

Preclinical Research in Glycogen Storage Diseases: A Comprehensive Review of Current Animal Models.

Almodóvar-Payá A, Villarreal-Salazar M, de Luna N, Nogales-Gadea G, Real-Martínez A, Andreu AL, Martín MA, Arenas J, Lucia A, Vissing J, Krag T, Pinós T. Almodóvar-Payá A, et al. Int J Mol Sci. 2020 Dec 17;21(24):9621. doi: 10.3390/ijms21249621. Int J Mol Sci. 2020. PMID: 33348688 Free PMC article. Review.

10

In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.

Landau DJ, Brooks ED, Perez-Pinera P, Amarasekara H, Mefferd A, Li S, Bird A, Gersbach CA, Koeberl DD. Landau DJ, et al. Mol Ther. 2016 Apr;24(4):697-706. doi: 10.1038/mt.2016.35. Epub 2016 Feb 11. Mol Ther. 2016. PMID: 26865405 Free PMC article.

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