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Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation
Emanuela Gussoni, … , Louis M. Kunkel, Kenneth Weinberg
Emanuela Gussoni, … , Louis M. Kunkel, Kenneth Weinberg
Published September 15, 2002
Citation Information: J Clin Invest. 2002;110(6):807-814. https://doi.org/10.1172/JCI16098.
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Article

Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation

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Abstract

Research Article

Authors

Emanuela Gussoni, Richard R. Bennett, Kristina R. Muskiewicz, Todd Meyerrose, Jan A. Nolta, Irene Gilgoff, James Stein, Yiu-mo Chan, Hart G. Lidov, Carsten G. Bönnemann, Arpad von Moers, Glenn E. Morris, Johan T. den Dunnen, Jeffrey S. Chamberlain, Louis M. Kunkel, Kenneth Weinberg

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Figure 5

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Analysis of dystrophin transcripts expressed in DMD-BMT1’s muscle. (a) U...
Analysis of dystrophin transcripts expressed in DMD-BMT1’s muscle. (a) U43–47 is unaffected control cDNA amplified with primers C43F-C47R; D43–47 and D43–48 are DMD-BMT1’s cDNA amplified using primers C43F-C47R or C43F-C48R, respectively. D43-48 shows the presence of three transcripts: del45 (655 bp), 43-46 (507 bp), and 44-48 (357 bp). (b) Schematic diagram of dystrophin transcripts amplified by RT-PCR and sequenced in DMD-BMT1. WT, wild type. (c) Amplification of full-length, donor-derived dystrophin in DMD-BMT1 (D43–45, D45–46) and in an unaffected control (U43–45, U45–46). (d) Quantitative RT-PCR analysis on total cDNA derived from DMD-BMT1 skeletal muscle. The cDNA was amplified using primers specific for the 45del isoform (dark blue), 44-48 in-frame truncated dystrophin (light blue), 43-46 in-frame truncated dystrophin (pink), and wild-type dystrophin (green). ΔRn, Δ reaction.

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