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The role of cell transplantation in modifying the course of limb girdle muscular dystrophy: a longitudinal 5-year study

Authors Sharma A, Sane H, Gokulchandran N, Gandhi S, Bhovad P, Khopkar D, Paranjape A, Bhagwanani K, Badhe P

Received 25 July 2014

Accepted for publication 23 September 2014

Published 14 September 2015 Volume 2015:5 Pages 93—102

DOI https://doi.org/10.2147/DNND.S71775

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Editor who approved publication: Dr Thomas Müller


Alok Sharma,1 Hemangi Sane,2 Nandini Gokulchandran,1 Sushant Gandhi,3 Pradnya Bhovad,3 Dipti Khopkar,2 Amruta Paranjape,2 Khushboo Bhagwanani,3 Prerna Badhe1

1Department of Medical Services and Clinical Research, 2Department of Research and Development, 3Department of Neurorehabilitation, NeuroGen Brain and Spine Institute, Stemasia Hospital and Research Centre, Nerul, Navi Mumbai, Maharashtra, India

Abstract: Limb girdle muscular dystrophy (LGMD), a group of progressive degenerative disorders, causes functional limitation affecting the quality of life. Cell therapy is being widely explored and preliminary studies have shown beneficial effects. Cell therapy induces trophic-factors release, angiogenesis, anti-inflammation, and protein synthesis, which helps in the reparative process at the microcellular level. In this 5-year longitudinal study, the effect of autologous bone marrow mononuclear cells is studied on the natural course of 65 patients with LGMD. Functional Independence Measure and manual muscle testing showed statistically significant improvement, post-cell transplantation. The key finding of this study was demonstration of a plateau phase in the disease progression of the patients. No adverse events were noted. Autologous bone marrow mononuclear cells may be a novel, safe, and effective treatment approach to control the rate of progression of LGMD, thus improving the functional outcomes. Further randomized controlled trials are required.

Keywords: cell therapy, autologous, bone marrow

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