LACK OF MESSENGER-RNA AND DYSTROPHIN EXPRESSION IN DMD PATIENTS 3 MONTHS AFTER MYOBLAST TRANSFER

被引:49
作者
MORANDI, L
BERNASCONI, P
GEBBIA, M
MORA, M
CROSTI, F
MANTEGAZZA, R
CORNELIO, F
机构
[1] Department of Neuromuscular Diseases, Istituto Nazionale Neurologico C. Besta, 20133 Milan
关键词
DMD; MYOBLAST TRANSFER; DYSTROPHIN MESSENGER-RNA;
D O I
10.1016/0960-8966(94)00070-P
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
We report our experience on myoblast transplantation in three Duchenne muscular dystrophy patients. Pure myoblasts (55 x 10(6) per patient) from HLA-matched donors, were injected into a tibialis anterior and the controlateral muscle was sham injected. Three months after transplantation, biopsies from the injected muscles were negative for dystrophin expression by immunocytochemistry. Reverse transcriptase-PCR (RT-PCR) failed to amplify any fragments of the deleted regions. This result confirms that myoblast transplantation is feasible, although the efficacy of this therapeutic approach is poor.
引用
收藏
页码:291 / 295
页数:5
相关论文
共 20 条
[1]   MONOCLONAL-ANTIBODY ANALYSIS OF MONONUCLEAR-CELLS IN MYOPATHIES .1. QUANTITATION OF SUBSETS ACCORDING TO DIAGNOSIS AND SITES OF ACCUMULATION AND DEMONSTRATION AND COUNTS OF MUSCLE-FIBERS INVADED BY T-CELLS [J].
ARAHATA, K ;
ENGEL, AG .
ANNALS OF NEUROLOGY, 1984, 16 (02) :193-208
[2]  
BEGGS AH, 1990, HUM GENET, V86, P45
[3]   ISOLATION AND CHARACTERIZATION OF HUMAN-MUSCLE CELLS [J].
BLAU, HM ;
WEBSTER, C .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA-BIOLOGICAL SCIENCES, 1981, 78 (09) :5623-5627
[4]  
CHAMBERLAIN JS, 1990, GUIDE METHODS APPLIC
[5]  
CHOMCZYNSKI P, 1987, ANAL BIOCHEM, V162, P156, DOI 10.1016/0003-2697(87)90021-2
[6]   MUSCLE-FIBER DEGENERATION AND NECROSIS IN MUSCULAR-DYSTROPHY AND OTHER MUSCLE DISEASES - CYTOCHEMICAL AND IMMUNOCYTOCHEMICAL DATA [J].
CORNELIO, F ;
DONES, I .
ANNALS OF NEUROLOGY, 1984, 16 (06) :694-701
[7]  
COVONE AE, 1992, AM J HUM GENET, V51, P675
[8]   NORMAL DYSTROPHIN TRANSCRIPTS DETECTED IN DUCHENNE MUSCULAR-DYSTROPHY PATIENTS AFTER MYOBLAST TRANSPLANTATION [J].
GUSSONI, E ;
PAVLATH, GK ;
LANCTOT, AM ;
SHARMA, KR ;
MILLER, RG ;
STEINMAN, L ;
BLAU, HM .
NATURE, 1992, 356 (6368) :435-438
[9]   HUMAN MYOBLAST TRANSPLANTATION - PRELIMINARY-RESULTS OF 4 CASES [J].
HUARD, J ;
BOUCHARD, JP ;
ROY, R ;
MALOUIN, F ;
DANSEREAU, G ;
LABRECQUE, C ;
ALBERT, N ;
RICHARDS, CL ;
LEMIEUX, B ;
TREMBLAY, JP .
MUSCLE & NERVE, 1992, 15 (05) :550-560
[10]   MYOBLAST TRANSFER IN DUCHENNE MUSCULAR-DYSTROPHY [J].
KARPATI, G ;
AJDUKOVIC, D ;
ARNOLD, D ;
GLEDHILL, RB ;
GUTTMANN, R ;
HOLLAND, P ;
KOCH, PA ;
SHOUBRIDGE, E ;
SPENCE, D ;
VANASSE, M ;
WATTERS, GV ;
ABRAHAMOWICZ, M ;
DUFF, C ;
WORTON, RG .
ANNALS OF NEUROLOGY, 1993, 34 (01) :8-17