Thöny B - Search Results

1

State-of-the-art 2023 on gene therapy for phenylketonuria.

Martinez M, Harding CO, Schwank G, Thöny B. Martinez M, et al. Among authors: thony b. J Inherit Metab Dis. 2024 Jan;47(1):80-92. doi: 10.1002/jimd.12651. Epub 2023 Aug 3. J Inherit Metab Dis. 2024. PMID: 37401651 Review.

2

State-of-the-art 2003 on PKU gene therapy.

Ding Z, Harding CO, Thöny B. Ding Z, et al. Among authors: thony b. Mol Genet Metab. 2004 Jan;81(1):3-8. doi: 10.1016/j.ymgme.2003.09.010. Mol Genet Metab. 2004. PMID: 14728985 Free PMC article. Review.

3

The fate of intravenously administered tetrahydrobiopterin and its implications for heterologous gene therapy of phenylketonuria.

Harding CO, Neff M, Wild K, Jones K, Elzaouk L, Thöny B, Milstien S. Harding CO, et al. Among authors: thony b. Mol Genet Metab. 2004 Jan;81(1):52-7. doi: 10.1016/j.ymgme.2003.10.002. Mol Genet Metab. 2004. PMID: 14728991 Free PMC article.

4

Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system.

Ding Z, Harding CO, Rebuffat A, Elzaouk L, Wolff JA, Thöny B. Ding Z, et al. Among authors: thony b. Mol Ther. 2008 Apr;16(4):673-81. doi: 10.1038/mt.2008.17. Epub 2008 Mar 11. Mol Ther. 2008. PMID: 18362925 Free PMC article.

5

Comparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuria.

Rebuffat A, Harding CO, Ding Z, Thöny B. Rebuffat A, et al. Among authors: thony b. Hum Gene Ther. 2010 Apr;21(4):463-77. doi: 10.1089/hum.2009.127. Hum Gene Ther. 2010. PMID: 19916803 Free PMC article.

6

Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle.

Thöny B. Thöny B. J Inherit Metab Dis. 2010 Dec;33(6):677-80. doi: 10.1007/s10545-010-9044-3. Epub 2010 Feb 12. J Inherit Metab Dis. 2010. PMID: 20151201 Review.

7

Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver.

Viecelli HM, Harbottle RP, Wong SP, Schlegel A, Chuah MK, VandenDriessche T, Harding CO, Thöny B. Viecelli HM, et al. Among authors: thony b. Hepatology. 2014 Sep;60(3):1035-43. doi: 10.1002/hep.27104. Epub 2014 Jul 29. Hepatology. 2014. PMID: 24585515 Free PMC article.

8

A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver.

Gallego-Villar L, Viecelli HM, Pérez B, Harding CO, Ugarte M, Thöny B, Desviat LR. Gallego-Villar L, et al. Among authors: thony b. Mol Ther Nucleic Acids. 2014 Sep 16;3(9):e193. doi: 10.1038/mtna.2014.44. Mol Ther Nucleic Acids. 2014. PMID: 25226162 Free PMC article.

9

High dose sapropterin dihydrochloride therapy improves monoamine neurotransmitter turnover in murine phenylketonuria (PKU).

Winn SR, Scherer T, Thöny B, Harding CO. Winn SR, et al. Among authors: thony b. Mol Genet Metab. 2016 Jan;117(1):5-11. doi: 10.1016/j.ymgme.2015.11.012. Epub 2015 Nov 26. Mol Genet Metab. 2016. PMID: 26653793 Free PMC article.

10

Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter.

Grisch-Chan HM, Schlegel A, Scherer T, Allegri G, Heidelberger R, Tsikrika P, Schmeer M, Schleef M, Harding CO, Häberle J, Thöny B. Grisch-Chan HM, et al. Among authors: thony b. Mol Ther Nucleic Acids. 2017 Jun 16;7:339-349. doi: 10.1016/j.omtn.2017.04.013. Epub 2017 Apr 20. Mol Ther Nucleic Acids. 2017. PMID: 28624210 Free PMC article.

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