Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients

Yvan Torrente, M. Belicchi, C. Marchesi, G. D'Antona, F. Cogiamanian, F. Pisati, M. Gavina, R. Giordano, R. Tonlorenzi, G. Fagiolari, C. Lamperti, L. Porretti, R. Lopa, M. Sampaolesi, L. Vicentini, N. Grimoldi, F. Tiberio, V. Songa, P. Baratta, A. PrelleL. Forzenigo, M. Guglieri, O. Pansarasa, C. Rinaldi, V. Mouly, G. S. Butler-Browne, G. P. Comi, P. Biondetti, M. Moggio, S. M. Gaini, N. Stocchetti, A. Priori, M. G. D'Angelo, A. Turconi, R. Bottinelli, G. Cossu, P. Rebulla, N. Bresolin

Research output: Contribution to journalArticle

Abstract

Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive muscle disease due to defect on the gene encoding dystrophin. The lack of a functional dystrophin in muscles results in the fragility of the muscle fiber membrane with progressive muscle weakness and premature death. There is no cure for DMD and current treatment options focus primarily on respiratory assistance, comfort care, and delaying the loss of ambulation. Recent works support the idea that stem cells can contribute to muscle repair as well as to replenishment of the satellite cell pool. Here we tested the safety of autologous transplantation of muscle-derived CD133+ cells in eight boys with Duchenne muscular dystrophy in a 7-month, double-blind phase I clinical trial. Stem cell safety was tested by measuring muscle strength and evaluating muscle structures with MRI and histological analysis. Timed cardiac and pulmonary function tests were secondary outcome measures. No local or systemic side effects were observed in all treated DMD patients. Treated patients had an increased ratio of capillary per muscle fibers with a switch from slow to fast myosin-positive myofibers.

Original languageEnglish
Pages (from-to)563-577
Number of pages15
JournalCell Transplantation
Volume16
Issue number6
Publication statusPublished - 2007

Fingerprint

Autologous Transplantation
Stem cells
Muscle
Stem Cells
Duchenne Muscular Dystrophy
Muscles
Dystrophin
Heart Function Tests
Safety
Clinical Trials, Phase I
Premature Mortality
Respiratory Function Tests
Muscle Weakness
Muscle Strength
Myosins
Gene encoding
Walking
Fibers
Magnetic resonance imaging
Outcome Assessment (Health Care)

Keywords

  • Autologous stem cell transplantation
  • CD133
  • Muscular dystrophy

ASJC Scopus subject areas

  • Cell Biology
  • Transplantation

Cite this

Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients. / Torrente, Yvan; Belicchi, M.; Marchesi, C.; D'Antona, G.; Cogiamanian, F.; Pisati, F.; Gavina, M.; Giordano, R.; Tonlorenzi, R.; Fagiolari, G.; Lamperti, C.; Porretti, L.; Lopa, R.; Sampaolesi, M.; Vicentini, L.; Grimoldi, N.; Tiberio, F.; Songa, V.; Baratta, P.; Prelle, A.; Forzenigo, L.; Guglieri, M.; Pansarasa, O.; Rinaldi, C.; Mouly, V.; Butler-Browne, G. S.; Comi, G. P.; Biondetti, P.; Moggio, M.; Gaini, S. M.; Stocchetti, N.; Priori, A.; D'Angelo, M. G.; Turconi, A.; Bottinelli, R.; Cossu, G.; Rebulla, P.; Bresolin, N.

In: Cell Transplantation, Vol. 16, No. 6, 2007, p. 563-577.

Research output: Contribution to journalArticle

Torrente, Y, Belicchi, M, Marchesi, C, D'Antona, G, Cogiamanian, F, Pisati, F, Gavina, M, Giordano, R, Tonlorenzi, R, Fagiolari, G, Lamperti, C, Porretti, L, Lopa, R, Sampaolesi, M, Vicentini, L, Grimoldi, N, Tiberio, F, Songa, V, Baratta, P, Prelle, A, Forzenigo, L, Guglieri, M, Pansarasa, O, Rinaldi, C, Mouly, V, Butler-Browne, GS, Comi, GP, Biondetti, P, Moggio, M, Gaini, SM, Stocchetti, N, Priori, A, D'Angelo, MG, Turconi, A, Bottinelli, R, Cossu, G, Rebulla, P & Bresolin, N 2007, 'Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients', Cell Transplantation, vol. 16, no. 6, pp. 563-577.
Torrente, Yvan ; Belicchi, M. ; Marchesi, C. ; D'Antona, G. ; Cogiamanian, F. ; Pisati, F. ; Gavina, M. ; Giordano, R. ; Tonlorenzi, R. ; Fagiolari, G. ; Lamperti, C. ; Porretti, L. ; Lopa, R. ; Sampaolesi, M. ; Vicentini, L. ; Grimoldi, N. ; Tiberio, F. ; Songa, V. ; Baratta, P. ; Prelle, A. ; Forzenigo, L. ; Guglieri, M. ; Pansarasa, O. ; Rinaldi, C. ; Mouly, V. ; Butler-Browne, G. S. ; Comi, G. P. ; Biondetti, P. ; Moggio, M. ; Gaini, S. M. ; Stocchetti, N. ; Priori, A. ; D'Angelo, M. G. ; Turconi, A. ; Bottinelli, R. ; Cossu, G. ; Rebulla, P. ; Bresolin, N. / Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients. In: Cell Transplantation. 2007 ; Vol. 16, No. 6. pp. 563-577.
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T1 - Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients

AU - Torrente, Yvan

AU - Belicchi, M.

AU - Marchesi, C.

AU - D'Antona, G.

AU - Cogiamanian, F.

AU - Pisati, F.

AU - Gavina, M.

AU - Giordano, R.

AU - Tonlorenzi, R.

AU - Fagiolari, G.

AU - Lamperti, C.

AU - Porretti, L.

AU - Lopa, R.

AU - Sampaolesi, M.

AU - Vicentini, L.

AU - Grimoldi, N.

AU - Tiberio, F.

AU - Songa, V.

AU - Baratta, P.

AU - Prelle, A.

AU - Forzenigo, L.

AU - Guglieri, M.

AU - Pansarasa, O.

AU - Rinaldi, C.

AU - Mouly, V.

AU - Butler-Browne, G. S.

AU - Comi, G. P.

AU - Biondetti, P.

AU - Moggio, M.

AU - Gaini, S. M.

AU - Stocchetti, N.

AU - Priori, A.

AU - D'Angelo, M. G.

AU - Turconi, A.

AU - Bottinelli, R.

AU - Cossu, G.

AU - Rebulla, P.

AU - Bresolin, N.

PY - 2007

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N2 - Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive muscle disease due to defect on the gene encoding dystrophin. The lack of a functional dystrophin in muscles results in the fragility of the muscle fiber membrane with progressive muscle weakness and premature death. There is no cure for DMD and current treatment options focus primarily on respiratory assistance, comfort care, and delaying the loss of ambulation. Recent works support the idea that stem cells can contribute to muscle repair as well as to replenishment of the satellite cell pool. Here we tested the safety of autologous transplantation of muscle-derived CD133+ cells in eight boys with Duchenne muscular dystrophy in a 7-month, double-blind phase I clinical trial. Stem cell safety was tested by measuring muscle strength and evaluating muscle structures with MRI and histological analysis. Timed cardiac and pulmonary function tests were secondary outcome measures. No local or systemic side effects were observed in all treated DMD patients. Treated patients had an increased ratio of capillary per muscle fibers with a switch from slow to fast myosin-positive myofibers.

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KW - Autologous stem cell transplantation

KW - CD133

KW - Muscular dystrophy

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