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Martier R, Sogorb-Gonzalez M, Stricker-Shaver J, et al. Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease. Mol Ther Methods Clin Dev. 2019;15:343-358doi: 10.1016/j.omtm.2019.10.008.
Martier, R., Sogorb-Gonzalez, M., Stricker-Shaver, J., Hübener-Schmid, J., Keskin, S., Klima, J., Toonen, L. J., Juhas, S., Juhasova, J., Ellederova, Z., Motlik, J., Haas, E., van Deventer, S., Konstantinova, P., Nguyen, H. P., & Evers, M. M. (2019). Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease. Molecular therapy. Methods & clinical development, 15343-358. https://doi.org/10.1016/j.omtm.2019.10.008
Martier, Raygene, et al. "Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease." Molecular therapy. Methods & clinical development vol. 15 (2019): 343-358. doi: https://doi.org/10.1016/j.omtm.2019.10.008
Martier R, Sogorb-Gonzalez M, Stricker-Shaver J, Hübener-Schmid J, Keskin S, Klima J, Toonen LJ, Juhas S, Juhasova J, Ellederova Z, Motlik J, Haas E, van Deventer S, Konstantinova P, Nguyen HP, Evers MM. Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease. Mol Ther Methods Clin Dev. 2019 Oct 28;15:343-358. doi: 10.1016/j.omtm.2019.10.008. eCollection 2019 Dec 13. PMID: 31828177; PMCID: PMC6889651.
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Mol Ther Methods Clin Dev. 2019 Oct 28;15:343-358. doi: 10.1016/j.omtm.2019.10.008. eCollection 2019 Dec 13.

Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease.

Molecular therapy. Methods & clinical development

Raygene Martier, Marina Sogorb-Gonzalez, Janice Stricker-Shaver, Jeannette Hübener-Schmid, Sonay Keskin, Jiri Klima, Lodewijk J Toonen, Stefan Juhas, Jana Juhasova, Zdenka Ellederova, Jan Motlik, Eva Haas, Sander van Deventer, Pavlina Konstantinova, Huu Phuc Nguyen, Melvin M Evers

Affiliations

  1. Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
  2. Department of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, the Netherlands.
  3. Institute of Medical Genetics and Applied Genomics, University of Tuebingen, Tuebingen, Germany.
  4. Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  5. Department of Human Genetics, Medical Faculty, Ruhr University Bochum, Bochum, Germany.

PMID: 31828177 PMCID: PMC6889651 DOI: 10.1016/j.omtm.2019.10.008

Abstract

Spinocerebellar ataxia type 3 (SCA3), or Machado-Joseph disease (MJD), is a progressive neurodegenerative disorder caused by a CAG expansion in the

© 2019 The Author(s).

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