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Year Number of Results
2002 1
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98 results

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Page 1
Gene therapy for glycogen storage diseases.
Koeberl DD, Koch RL, Lim JA, Brooks ED, Arnson BD, Sun B, Kishnani PS. Koeberl DD, et al. J Inherit Metab Dis. 2024 Jan;47(1):93-118. doi: 10.1002/jimd.12654. Epub 2023 Jul 27. J Inherit Metab Dis. 2024. PMID: 37421310 Review.
IRF2BPL Is Associated with Neurological Phenotypes.
Marcogliese PC, Shashi V, Spillmann RC, Stong N, Rosenfeld JA, Koenig MK, Martínez-Agosto JA, Herzog M, Chen AH, Dickson PI, Lin HJ, Vera MU, Salamon N, Graham JM Jr, Ortiz D, Infante E, Steyaert W, Dermaut B, Poppe B, Chung HL, Zuo Z, Lee PT, Kanca O, Xia F, Yang Y, Smith EC, Jasien J, Kansagra S, Spiridigliozzi G, El-Dairi M, Lark R, Riley K, Koeberl DD, Golden-Grant K; Program for Undiagnosed Diseases (UD-PrOZA); Undiagnosed Diseases Network; Yamamoto S, Wangler MF, Mirzaa G, Hemelsoet D, Lee B, Nelson SF, Goldstein DB, Bellen HJ, Pena LDM. Marcogliese PC, et al. Among authors: koeberl dd. Am J Hum Genet. 2018 Aug 2;103(2):245-260. doi: 10.1016/j.ajhg.2018.07.006. Epub 2018 Jul 26. Am J Hum Genet. 2018. PMID: 30057031 Free PMC article.
Gene therapy for glycogen storage diseases.
Kishnani PS, Sun B, Koeberl DD. Kishnani PS, et al. Among authors: koeberl dd. Hum Mol Genet. 2019 Oct 1;28(R1):R31-R41. doi: 10.1093/hmg/ddz133. Hum Mol Genet. 2019. PMID: 31227835 Free PMC article. Review.
Staying on target in gene and cell therapy.
Bricker-Anthony R, Koeberl DD, Lipshutz GS, Perna F. Bricker-Anthony R, et al. Among authors: koeberl dd. Mol Ther. 2024 Sep 4;32(9):2801-2802. doi: 10.1016/j.ymthe.2024.08.010. Epub 2024 Aug 23. Mol Ther. 2024. PMID: 39178850 No abstract available.
Liver depot gene therapy for Pompe disease.
Kishnani PS, Koeberl DD. Kishnani PS, et al. Among authors: koeberl dd. Ann Transl Med. 2019 Jul;7(13):288. doi: 10.21037/atm.2019.05.02. Ann Transl Med. 2019. PMID: 31392200 Free PMC article. Review.
Phase I study of liver depot gene therapy in late-onset Pompe disease.
Smith EC, Hopkins S, Case LE, Xu M, Walters C, Dearmey S, Han SO, Spears TG, Chichester JA, Bossen EH, Hornik CP, Cohen JL, Bali D, Kishnani PS, Koeberl DD. Smith EC, et al. Among authors: koeberl dd. Mol Ther. 2023 Jul 5;31(7):1994-2004. doi: 10.1016/j.ymthe.2023.02.014. Epub 2023 Feb 18. Mol Ther. 2023. PMID: 36805083 Free PMC article. Clinical Trial.
98 results