PMID- 12235824
OWN - NLM
STAT- MEDLINE
DCOM- 20021108
LR  - 20121115
IS  - 0009-918X (Print)
IS  - 0009-918X (Linking)
VI  - 41
IP  - 12
DP  - 2001 Dec
TI  - [Development of new therapy on muscular dystrophy].
PG  - 1154-6
AB  - Duchenne muscular dystrophy (DMD) is an X-linked, lethal disorder caused by a 
      defect in the DMD gene. We have previously reported that micro-dystrophins, which 
      have large deletions in rod repeat domain, successfully localize at the 
      sarcolemma and stabilize dystroglycan-sarcoglycan complex in dystrophin-deficient 
      mdx muscle. However, expression of a 3.7-kb micro-dystrophin cDNA, having only 
      one rod repeat showed no effect on dystrophic phenotype. Further transgenic 
      experiments are carrying to seek a functional but small-sized micro-dystrophin 
      cDNA, which can be accommodated into Adeno-associated virus (AAV) vector. In 
      normal muscle, AAV-LacZ vector expresses stably beta-gal for a long period, 
      however, we noticed that immune response is evoked by AAV-LacZ vector in mdx 
      muscle. Therefore, for successful gene therapy, it is required to reduce immune 
      response against AAV-dystrophin vector and therapeutic proteins in mdx mice. We 
      have already reported that utrophin was up-regulated at the sarcolemma of mdx 
      mice, when a beta-galactosidase-expressing adenovirus vector, AxCALacZ was 
      injected into the skeletal muscle. Moreover, up-regulated utrophin mitigated 
      dystrophic phenotypes. Up-regulation of utrophin was induced by inflammatory 
      response against adenovirus vector-mediated gene transfer and this up-regulation 
      is one of promising tools for treatment of DMD.
FAU - Takeda, S
AU  - Takeda S
LA  - jpn
PT  - English Abstract
PT  - Journal Article
PT  - Review
PL  - Japan
TA  - Rinsho Shinkeigaku
JT  - Rinsho shinkeigaku = Clinical neurology
JID - 0417466
RN  - 0 (Cytoskeletal Proteins)
RN  - 0 (Dystrophin)
RN  - 0 (Membrane Proteins)
RN  - 0 (Utrn protein, mouse)
RN  - 0 (Utrophin)
SB  - IM
MH  - Animals
MH  - Cytoskeletal Proteins/genetics/metabolism
MH  - Drug Design
MH  - Dystrophin/genetics
MH  - Genetic Therapy
MH  - Humans
MH  - Membrane Proteins/genetics/metabolism
MH  - Mice
MH  - Muscular Dystrophies/*therapy
MH  - Stem Cell Transplantation
MH  - Up-Regulation
MH  - Utrophin
RF  - 10
EDAT- 2002/09/19 10:00
MHDA- 2002/11/26 04:00
CRDT- 2002/09/19 10:00
PHST- 2002/09/19 10:00 [pubmed]
PHST- 2002/11/26 04:00 [medline]
PHST- 2002/09/19 10:00 [entrez]
PST - ppublish
SO  - Rinsho Shinkeigaku. 2001 Dec;41(12):1154-6.