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Table representation of search results timeline featuring number of search results per year.
Year | Number of Results |
---|---|
2021 | 3 |
2022 | 1 |
2023 | 2 |
2024 | 1 |
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6 results
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Page 1
Fetal gene therapy.
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.
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C1 Disease.
Hughes MP, Nelvagal HR, Coombe-Tennant O, Smith D, Smith C, Massaro G, Poupon-Bejuit L, Platt FM, Rahim AA.
Hughes MP, et al.
Cells. 2023 Jun 13;12(12):1619. doi: 10.3390/cells12121619.
Cells. 2023.
PMID: 37371089
Free PMC article.
Item in Clipboard
A GLP1 receptor agonist diabetes drug ameliorates neurodegeneration in a mouse model of infantile neurometabolic disease.
Poupon-Bejuit L, Hughes MP, Liu W, Geard A, Faour-Slika N, Whaler S, Massaro G, Rahim AA.
Poupon-Bejuit L, et al.
Sci Rep. 2022 Aug 15;12(1):13825. doi: 10.1038/s41598-022-17338-1.
Sci Rep. 2022.
PMID: 35970890
Free PMC article.
Item in Clipboard
Gene therapy effective in model of infantile parkinsonism.
Lemprière S.
Lemprière S.
Nat Rev Neurol. 2021 Jul;17(7):394. doi: 10.1038/s41582-021-00522-8.
Nat Rev Neurol. 2021.
PMID: 34083766
No abstract available.
Item in Clipboard
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.
Item in Clipboard
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
Massaro G, Geard AF, Liu W, Coombe-Tennant O, Waddington SN, Baruteau J, Gissen P, Rahim AA.
Massaro G, et al.
Biomolecules. 2021 Apr 20;11(4):611. doi: 10.3390/biom11040611.
Biomolecules. 2021.
PMID: 33924076
Free PMC article.
Review.
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