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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 and Tian, Zeng Min (2016) In Experimental and Therapeutic Medicine 12(2). p.661-666
Abstract

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... (More)

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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author
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Frameless stereotactic surgery, Neural stem cells, Primary torsion spasm, Torsion dystonia-1, Transplantation
in
Experimental and Therapeutic Medicine
volume
12
issue
2
pages
6 pages
publisher
Spandidos Publications Ltd
external identifiers
  • Scopus:84974711175
ISSN
1792-0981
DOI
10.3892/etm.2016.3392
language
English
LU publication?
no
id
566c9c06-36af-4c88-b6c3-287caa809c36
date added to LUP
2016-07-06 15:09:09
date last changed
2016-09-29 16:30:22
@misc{566c9c06-36af-4c88-b6c3-287caa809c36,
  abstract     = {<p>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.</p>},
  author       = {Ren, Wen Qing and Yin, Feng and Zhang, Jian Ning and Lu, Wang Sheng and Liang, Ying Kui and Adlerberth, Josefin and Tian, Zeng Min},
  issn         = {1792-0981},
  keyword      = {Frameless stereotactic surgery,Neural stem cells,Primary torsion spasm,Torsion dystonia-1,Transplantation},
  language     = {eng},
  month        = {08},
  number       = {2},
  pages        = {661--666},
  publisher    = {ARRAY(0x3691618)},
  series       = {Experimental and Therapeutic Medicine},
  title        = {Neural stem cell transplantation for the treatment of primary torsion dystonia : A case report},
  url          = {http://dx.doi.org/10.3892/etm.2016.3392},
  volume       = {12},
  year         = {2016},
}