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Year Number of Results
2017 3
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2021 2
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Page 1
Fetal gene therapy.
Waddington SN, Peranteau WH, Rahim AA, Boyle AK, Kurian MA, Gissen P, Chan JKY, David AL. Waddington SN, et al. J Inherit Metab Dis. 2024 Jan;47(1):192-210. doi: 10.1002/jimd.12659. Epub 2023 Aug 7. J Inherit Metab Dis. 2024. PMID: 37470194 Free PMC article. Review.
Three-Country Snapshot of Ornithine Transcarbamylase Deficiency.
Seker Yilmaz B, Baruteau J, Arslan N, Aydin HI, Barth M, Bozaci AE, Brassier A, Canda E, Cano A, Chronopoulou E, Connolly GM, Damaj L, Dawson C, Dobbelaere D, Douillard C, Eminoglu FT, Erdol S, Ersoy M, Fang S, Feillet F, Gokcay G, Goksoy E, Gorce M, Inci A, Kadioglu B, Kardas F, Kasapkara CS, Kilic Yildirim G, Kor D, Kose M, Marelli C, Mundy H, O'Sullivan S, Ozturk Hismi B, Ramachandran R, Roubertie A, Sanlilar M, Schiff M, Sreekantam S, Stepien KM, Uzun Unal O, Yildiz Y, Zubarioglu T, Gissen P. Seker Yilmaz B, et al. Life (Basel). 2022 Oct 27;12(11):1721. doi: 10.3390/life12111721. Life (Basel). 2022. PMID: 36362876 Free PMC article.
Gene therapies targeting the liver.
Cozmescu AC, Counsell J, Gissen P. Cozmescu AC, et al. J Hepatol. 2021 Jan;74(1):235-236. doi: 10.1016/j.jhep.2020.08.003. Epub 2020 Oct 16. J Hepatol. 2021. PMID: 33071009 No abstract available.
Liver neoplasms in methylmalonic aciduria: An emerging complication.
Forny P, Hochuli M, Rahman Y, Deheragoda M, Weber A, Baruteau J, Grunewald S. Forny P, et al. J Inherit Metab Dis. 2019 Sep;42(5):793-802. doi: 10.1002/jimd.12143. Epub 2019 Jul 17. J Inherit Metab Dis. 2019. PMID: 31260114 Free article.
Fetal gene therapy for neurodegenerative disease of infants.
Massaro G, Mattar CNZ, Wong AMS, Sirka E, Buckley SMK, Herbert BR, Karlsson S, Perocheau DP, Burke D, Heales S, Richard-Londt A, Brandner S, Huebecker M, Priestman DA, Platt FM, Mills K, Biswas A, Cooper JD, Chan JKY, Cheng SH, Waddington SN, Rahim AA. Massaro G, et al. Nat Med. 2018 Sep;24(9):1317-1323. doi: 10.1038/s41591-018-0106-7. Epub 2018 Jul 16. Nat Med. 2018. PMID: 30013199 Free PMC article.
21 results