Authors: Torrente, Y.¹; D'Angelo, M.G.²; Li, Z.³; DelBo, R.⁴; Corti, S.²; Mericskay, M.³; DeLiso, A.²; Fassati, A.⁵; Paulin, D.³; Comi, G.P.¹; Scarlato, G.¹; Bresolin, N.¹

Source: Human Molecular Genetics, Volume 9, Number 12, 22 July 2000 , pp. 1843-1852(10)

Publisher: Oxford University Press

Abstract:

Duchenne muscular dystrophy (DMD) is a lethal recessive disease caused by the absence of dystrophin in skeletal muscle, heart and other tissues. No cure is available at present for DMD. Here we describe a new strategy for the correction of dystrophin deficiency based on the transplantation of normal somite-derived cells into mdxmouse embryos. Somite-derived cells were isolated from E11.5 transgenic mouse embryos expressing the LacZ gene under the control of the muscle-specific desmin promoter and injected into the uterine circulation of pregnant mdx mice at gestational days E11.5-E17. Approximately 30% of the injected mdx embryos survived the procedure. Donor somite-derived cells were able to cross the placenta and migrate into host embryonic tissues. The pattern of donor cell distribution in host tissues depended on the gestational age of the transplanted embryos. Cells were found in hindlimb muscles, diaphragm, heart and ribs in E11.5 treated embryos and in the skull, ribs, vertebrae and lung of E15-E17 treated embryos. Normal dystrophin transcripts were detected in muscle and bone by RT-PCR. Histochemical analysis showed co-localization of LacZ and dystrophin expression in 5% of soleus and quadriceps muscle fibres and in 4% of heart myocytes of two of seven 8-week-old treated mdx mice.

Document Type: Research article

Affiliations: 1: IRCCS Ospedale Maggiore Policlinico, Milan, Italy, 2: Centro Dino Ferrari, Institute of Clinical Neurology, University of Milan, Milan, Italy, 3: Université Paris 7, Case 7136, 2 place Jussieu, 75251 Paris, France and 4: IRCCS Eugenio Medea, Bosisio Parini, Italy, 5: Wohl Virion Centre, Windeyer Institute, University College London, 46 Cleveland Street, London W1P 6BD, UK

Publication date: 2000-07-22

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• Human Molecular Genetics concentrates on full-length research papers covering a wide range of topics in all aspects of human molecular genetics.

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• In this Subject: Biology ,  Biotechnology ,  Pathology ,  Genetics
• By this author: Torrente, Y. ;  D'Angelo, M.G. ;  Li, Z. ;  DelBo, R. ;  Corti, S. ;  Mericskay, M. ;  DeLiso, A. ;  Fassati, A. ;  Paulin, D. ;  Comi, G.P. ;  Scarlato, G. ;  Bresolin, N.

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