In:
Current Gene Therapy, Bentham Science Publishers Ltd., Vol. 23, No. 4 ( 2023-08), p. 304-315
Abstract:
Duchenne Muscular Dystrophy (DMD) results in a deficiency of dystrophin expression
in patient muscle fibers, leading to progressive muscle degeneration. Treatment of DMD has undertaken current transformation with the advancement of novel gene therapy and molecular biology
techniques, which are secure, well-tolerated, and effective therapeutic approaches. Introduction: DMD gene therapies have mainly focused on young DMD patients as in vivo animal
model trials have been performed in 0–1-month DMD mice. However, it has not yet been answered how micro-dystrophin encoding lentiviral treatment affects Dystrophin expression and DMD symptoms
in 10-month mdx mice. Methods: We planned to integrate the micro-Dystrophin gene sequence into the muscle cells by viral
transfer, using micro-Dystrophin-encoding lentivirus to reduce the dystrophic pathology in late-stage dmd mice. The histopathological and physiological-functional regeneration activities of the lentiviralmicro-
Dystrophin gene therapy m ethods were compared, along with changes in temporal Dystrophin
expression and their functionality, toxicity, and gene expression level. Results: Here, we showed that the micro-dystrophin transgene transfers intramuscularly and intraperitoneally
in late-stage dmd-mdx-4cv mice restored dystrophin expression in the skeletal and cardiac muscle (p 〈 0.001). Furthermore, motor performance analysis, including hanging and tracking tests,
improved statistically significantly after the treatment (p 〈 0.05). Conclusion: Consequently, this study suggests that patients in the late stages of muscular dystrophy
can benefit from lentiviral micro-dystrophin gene therapies to present an improvement in dystrophic muscle pathology.
Type of Medium:
Online Resource
ISSN:
1566-5232
DOI:
10.2174/1566523223666230407091317
Language:
English
Publisher:
Bentham Science Publishers Ltd.
Publication Date:
2023
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