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Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report

Primary torsion dystonia (PTD) occurs due to a genetic mutation and often advances gradually. Currently, there is no therapy available that is able to inhibit progression. Neural stem cells (NSCs) are being investigated as potential therapies for neurodegenerative diseases, such as stroke and trauma...

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Autores principales: Ren, Wen-Qing, Yin, Feng, Zhang, Jian-Ning, Lu, Wang-Sheng, Liang, Ying-Kui, Adlerberth, Josefin, Tian, Zeng-Min
Formato: Online Artículo Texto
Lenguaje:English
Publicado: D.A. Spandidos 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4950735/
https://www.ncbi.nlm.nih.gov/pubmed/27446258
http://dx.doi.org/10.3892/etm.2016.3392
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author Ren, Wen-Qing
Yin, Feng
Zhang, Jian-Ning
Lu, Wang-Sheng
Liang, Ying-Kui
Adlerberth, Josefin
Tian, Zeng-Min
author_facet Ren, Wen-Qing
Yin, Feng
Zhang, Jian-Ning
Lu, Wang-Sheng
Liang, Ying-Kui
Adlerberth, Josefin
Tian, Zeng-Min
author_sort Ren, Wen-Qing
collection PubMed
description Primary torsion dystonia (PTD) occurs due to a genetic mutation and often advances gradually. Currently, there is no therapy available that is able to inhibit progression. Neural stem cells (NSCs) are being investigated as potential therapies for neurodegenerative diseases, such as stroke and trauma. The present study evaluated the clinical effectiveness of NSC transplantation in an 18-year-old male patient with PTD, to assess the ability of this therapy to inhibit PTD progression. Genetic testing of the patient revealed a mutation in the torsion dystonia-1 (DYT1) gene (907–909 delGAG). NSCs were bilaterally implanted in the globus pallidus of the patient through stereotactic surgery. Prior to surgery, the patient's Burke-Fahn-Marsden dystonia movement score (BFMDMS) was 21, which progressively decreased after surgery to 18, 17, 15 and 13 at 1, 2, 3 and 4 postoperative years, respectively. BFMDMS was improved by 38.1% over the 4 postoperative years. Although computed tomography and magnetic resonance imaging examinations showed no significant changes prior to and following surgery, postoperative brain positron emission tomography scans revealed increased glucose metabolism in the transplanted region. The clinical efficacy of NSC transplantation in this patient suggests its potential for the treatment of DYT1-positive patients with PTD.
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spelling pubmed-49507352016-07-21 Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report Ren, Wen-Qing Yin, Feng Zhang, Jian-Ning Lu, Wang-Sheng Liang, Ying-Kui Adlerberth, Josefin Tian, Zeng-Min Exp Ther Med Articles Primary torsion dystonia (PTD) occurs due to a genetic mutation and often advances gradually. Currently, there is no therapy available that is able to inhibit progression. Neural stem cells (NSCs) are being investigated as potential therapies for neurodegenerative diseases, such as stroke and trauma. The present study evaluated the clinical effectiveness of NSC transplantation in an 18-year-old male patient with PTD, to assess the ability of this therapy to inhibit PTD progression. Genetic testing of the patient revealed a mutation in the torsion dystonia-1 (DYT1) gene (907–909 delGAG). NSCs were bilaterally implanted in the globus pallidus of the patient through stereotactic surgery. Prior to surgery, the patient's Burke-Fahn-Marsden dystonia movement score (BFMDMS) was 21, which progressively decreased after surgery to 18, 17, 15 and 13 at 1, 2, 3 and 4 postoperative years, respectively. BFMDMS was improved by 38.1% over the 4 postoperative years. Although computed tomography and magnetic resonance imaging examinations showed no significant changes prior to and following surgery, postoperative brain positron emission tomography scans revealed increased glucose metabolism in the transplanted region. The clinical efficacy of NSC transplantation in this patient suggests its potential for the treatment of DYT1-positive patients with PTD. D.A. Spandidos 2016-08 2016-05-25 /pmc/articles/PMC4950735/ /pubmed/27446258 http://dx.doi.org/10.3892/etm.2016.3392 Text en Copyright: © Ren et al. This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License (https://creativecommons.org/licenses/by-nc-nd/4.0/) , which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.
spellingShingle Articles
Ren, Wen-Qing
Yin, Feng
Zhang, Jian-Ning
Lu, Wang-Sheng
Liang, Ying-Kui
Adlerberth, Josefin
Tian, Zeng-Min
Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report
title Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report
title_full Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report
title_fullStr Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report
title_full_unstemmed Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report
title_short Neural stem cell transplantation for the treatment of primary torsion dystonia: A case report
title_sort neural stem cell transplantation for the treatment of primary torsion dystonia: a case report
topic Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4950735/
https://www.ncbi.nlm.nih.gov/pubmed/27446258
http://dx.doi.org/10.3892/etm.2016.3392
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