32474930 31890731 36261499 30916397 34610166 30255460 30808235 34157136 36101819 35573684 31227835 31787497 31115117 31319709 30174654 - Search Results

Year	Number of Results
2018	2
2019	7
2020	3
2021	3
2022	3
2023	0

1

Emerging roles of autophagy in hepatic tumorigenesis and therapeutic strategies in glycogen storage disease type Ia: A review.

Cho JH, Weinstein DA, Lee YM. Cho JH, et al. J Inherit Metab Dis. 2021 Jan;44(1):118-128. doi: 10.1002/jimd.12267. Epub 2020 Jul 2. J Inherit Metab Dis. 2021. PMID: 32474930 Review.

2

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease.

Kang HR, Gjorgjieva M, Smith SN, Brooks ED, Chen Z, Burgess SM, Chandler RJ, Waskowicz LR, Grady KM, Li S, Mithieux G, Venditti CP, Rajas F, Koeberl DD. Kang HR, et al. Mol Ther Methods Clin Dev. 2019 Nov 11;15:383-391. doi: 10.1016/j.omtm.2019.10.016. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31890731 Free PMC article.

3

Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors.

Kim AY, Duerr FM, Phillips JN, Samulski RJ, Grieger JC, Goodrich LR. Kim AY, et al. Gene Ther. 2022 Oct 20. doi: 10.1038/s41434-022-00366-x. Online ahead of print. Gene Ther. 2022. PMID: 36261499

4

Exploiting epigenetics for the treatment of inborn errors of metabolism.

Rutten MGS, Rots MG, Oosterveer MH. Rutten MGS, et al. J Inherit Metab Dis. 2020 Jan;43(1):63-70. doi: 10.1002/jimd.12093. Epub 2019 Apr 22. J Inherit Metab Dis. 2020. PMID: 30916397 Free PMC article. Review.

5

Polyadenine insertion disrupting the G6PC1 gene in German Pinschers with glycogen storage disease type Ia (GSD1A).

Christen M, Reineking W, Beineke A, Jagannathan V, Baumgärtner W, Leeb T. Christen M, et al. Anim Genet. 2021 Dec;52(6):900-902. doi: 10.1111/age.13146. Epub 2021 Oct 5. Anim Genet. 2021. PMID: 34610166 Free PMC article. No abstract available.

6

Letter to the Editors: Concerning "Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia" by Lee et al.

Brooks ED, Kishnani PS, Koeberl DD. Brooks ED, et al. J Inherit Metab Dis. 2018 Nov;41(6):913-914. doi: 10.1007/s10545-018-0248-2. Epub 2018 Sep 25. J Inherit Metab Dis. 2018. PMID: 30255460 Free PMC article. No abstract available.

7

Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.

Guilbaud M, Devaux M, Couzinié C, Le Duff J, Toromanoff A, Vandamme C, Jaulin N, Gernoux G, Larcher T, Moullier P, Le Guiner C, Adjali O. Guilbaud M, et al. Hum Gene Ther. 2019 Jul;30(7):802-813. doi: 10.1089/hum.2018.234. Epub 2019 Apr 16. Hum Gene Ther. 2019. PMID: 30808235 Free PMC article.

8

Modeling Phenotypic Heterogeneity of Glycogen Storage Disease Type 1a Liver Disease in Mice by Somatic CRISPR/CRISPR-associated protein 9-Mediated Gene Editing.

Rutten MGS, Derks TGJ, Huijkman NCA, Bos T, Kloosterhuis NJ, van de Kolk KCWA, Wolters JC, Koster MH, Bongiovanni L, Thomas RE, de Bruin A, van de Sluis B, Oosterveer MH. Rutten MGS, et al. Hepatology. 2021 Nov;74(5):2491-2507. doi: 10.1002/hep.32022. Epub 2021 Aug 15. Hepatology. 2021. PMID: 34157136 Free PMC article.

9

Glycogen storage disease type 1a in the Ohio Amish.

Scott EM, Wenger OK, Robinson E, Colling K, Brown MF, Hershberger J, Radhakrishnan K. Scott EM, et al. JIMD Rep. 2022 Jun 21;63(5):453-461. doi: 10.1002/jmd2.12310. eCollection 2022 Sep. JIMD Rep. 2022. PMID: 36101819 Free PMC article.

10

Virally Mediated Connexin 26 Expression in Postnatal Scala Media Significantly and Transiently Preserves Hearing in Connexin 30 Null Mice.

Zhang L, Wang W, Kim SM, Wang J, Zhou B, Kong W, Zheng J, Lin X. Zhang L, et al. Front Cell Dev Biol. 2022 Apr 27;10:900416. doi: 10.3389/fcell.2022.900416. eCollection 2022. Front Cell Dev Biol. 2022. PMID: 35573684 Free PMC article.

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