PMID- 9288722
OWN - NLM
STAT- MEDLINE
DCOM- 19971023
LR  - 20220311
IS  - 1078-8956 (Print)
IS  - 1078-8956 (Linking)
VI  - 3
IP  - 9
DP  - 1997 Sep
TI  - The fate of individual myoblasts after transplantation into muscles of DMD 
      patients.
PG  - 970-7
AB  - Muscle biopsies from six patients with Duchenne muscular dystrophy (DMD) 
      participating in a myoblast transplantation clinical trial were reexamined using 
      a fluorescence in situ hybridization (FISH)-based method. Donor nuclei were 
      detected in all biopsies analyzed, including nine where no donor myoblasts were 
      previously thought to be present. In three patients, more than 10% of the 
      original number of donor cells were calculated as present 6 months after 
      implantation. Half of the detected donor nuclei were fused into host myofibers, 
      and of these, nearly 50% produced dystrophin. These findings demonstrate that 
      although donor myoblasts have persisted after injection, their microenvironment 
      influences whether they fuse and express dystrophin. Our methodology could be 
      used for developing new approaches to improve myoblast transfer efficacy and for 
      the analysis of future gene- and/or cell-based therapies of numerous genetic 
      disorders.
FAU - Gussoni, E
AU  - Gussoni E
AD  - Division of Genetics, Children's Hospital, Boston, Massachusetts 02115, USA.
FAU - Blau, H M
AU  - Blau HM
FAU - Kunkel, L M
AU  - Kunkel LM
LA  - eng
GR  - NS23740/NS/NINDS NIH HHS/United States
PT  - Clinical Trial
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Nat Med
JT  - Nature medicine
JID - 9502015
RN  - 0 (Dystrophin)
SB  - IM
MH  - Cell Count
MH  - Cell Fusion
MH  - Cell Nucleus/pathology
MH  - Cell Transplantation/adverse effects
MH  - Dystrophin/genetics/metabolism
MH  - Gene Expression
MH  - Humans
MH  - Immunohistochemistry
MH  - In Situ Hybridization, Fluorescence
MH  - Male
MH  - Muscles/metabolism/pathology/*transplantation
MH  - Muscular Dystrophies/metabolism/pathology/*therapy
MH  - T-Lymphocytes/immunology/pathology
MH  - Time Factors
MH  - Tissue Donors
EDAT- 1997/09/01 00:00
MHDA- 1997/09/01 00:01
CRDT- 1997/09/01 00:00
PHST- 1997/09/01 00:00 [pubmed]
PHST- 1997/09/01 00:01 [medline]
PHST- 1997/09/01 00:00 [entrez]
AID - 10.1038/nm0997-970 [doi]
PST - ppublish
SO  - Nat Med. 1997 Sep;3(9):970-7. doi: 10.1038/nm0997-970.