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ctt-journal > Mikhailov et al. (Abstract)

Mikhailov et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 12
doi: 10.3205/ctt-2011-No12-abstract22

© The Authors. This abstract is provided under the following license: Creative Commons Attribution 3.0 Unported

Abstract accepted for "5th Raisa Gorbacheva Memorial Meeting Hematopoietic Stem Cell Transplantation in Children and Adults", Saint Petersburg, Russia, September 18–20, 2011

Preliminary Program

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Bone marrow transplantation as method of treatment of mdx mice myodystrophy

Viacheslav M. Mikhailov1, Anastasia V. Sokolova1, Natalie A. Timonina2, Violeta V. Kravtsova2, Igor I. Krivoy2

1Institute of Cytology Russian Academy of Sciences, St. Petersburg, Russia; 2St. Petersburg State University, St. Petersburg, Russia

Correspondence: Viacheslav M. Mikhailov, Institute of Cytology RAS, 4, Tikhorecky avenue, St. Petersburg, 194064, Russia, E-mail: vmikhailov@spam is badmail.cytspb.rssi.ru


In the course of monogenic diseases stem cells contain mutant genes, which, when expressed in differentiated cells, cause the disturbance of the cell’s function and survival. The replacement of the mutant stem cells with wild stem cells is seen as radical approach to the treatment of monogenic diseases. Bone marrow transplantations are used to treat monogenic diseases β-thalassemia and sickle cell anemia. We present the results of treatment of the monogenic disease myodystrophy of mdx mice, the counterpart of human Duchenne myodystrophy, by C57BL/6 bone marrow transplantation.

After 3, 5, and 7.5 Gy X-ray irradiation, mdx mice were transplanted with syngeneic C57BL/6 mice bone marrow. Skeletal muscle fibers (MF) and diaphragm muscle fibers were studied for MF death, accumulation of MF without central nuclei, and for dystrophin synthesis. Synapses were analyzed by confocal microscopy after staining with α-Bungarothoxin–tetramethyl-rhodamine. The membrane resting potential of terminal plates (MRP) was measured by the microelectrode technique.

After irradiation with 5 and 7.5 Gy, bone marrow transplantation was not accompanied by a growth of dystrophin synthesis and by increasing of differentiation level. After 3 Gy irradiation there was growth of dystrophin-positive MF from 1.1% (before transplantation) up to 4.1% (2 months), 12.4% (4 months) and 27.6% (6 months after bone marrow transplantation). Diaphragm MFs were dystrophin-positive at 44%. The level of MF differentiation increased, MRP grew up from -75.1 мV up to level of normal C57BL/6 mice -80.7 мV.

Therefore the transplantation of wild type of bone marrow restores differentiation and electrogenesis of mdx mice skeletal MFs up to the level of normal animals.

Work was supported by RFBR Grant # 10-04-00970а and by RF MES contract № 02.740.11.0094.

Keywords: monogenic myodystrophy, mdx mice, bone marrow transplantation