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Year Number of Results
2017 2
2018 3
2019 6
2020 4
2021 2
2022 1
2023 2
2024 1

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17 results

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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 Review.
Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.
Cunningham SC, van Dijk EB, Zhu E, Sugden M, Mandwie M, Siew S, Devanapalli B, Tolun AA, Klein A, Wilson L, Aryamanesh N, Gissen P, Baruteau J, Bhattacharya K, Alexander IE. Cunningham SC, et al. Hum Gene Ther. 2023 Sep;34(17-18):917-926. doi: 10.1089/hum.2023.011. Epub 2023 Sep 11. Hum Gene Ther. 2023. PMID: 37350098
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 therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.
Ng J, Barral S, De La Fuente Barrigon C, Lignani G, Erdem FA, Wallings R, Privolizzi R, Rossignoli G, Alrashidi H, Heasman S, Meyer E, Ngoh A, Pope S, Karda R, Perocheau D, Baruteau J, Suff N, Antinao Diaz J, Schorge S, Vowles J, Marshall LR, Cowley SA, Sucic S, Freissmuth M, Counsell JR, Wade-Martins R, Heales SJR, Rahim AA, Bencze M, Waddington SN, Kurian MA. Ng J, et al. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564. Sci Transl Med. 2021. PMID: 34011628 Free PMC article.
An In Vitro Whole-Organ Liver Engineering for Testing of Genetic Therapies.
Lorvellec M, Pellegata AF, Maestri A, Turchetta C, Alvarez Mediavilla E, Shibuya S, Jones B, Scottoni F, Perocheau DP, Cozmescu AC, Delhove JM, Kysh D, Gjinovci A, Counsell JR, Heywood WE, Mills K, McKay TR, De Coppi P, Gissen P. Lorvellec M, et al. iScience. 2020 Nov 13;23(12):101808. doi: 10.1016/j.isci.2020.101808. eCollection 2020 Dec 18. iScience. 2020. PMID: 33305175 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.
17 results