What question did this study set out to answer?

Evaluate the feasibility, short-term protective efficacy, and long-term persistence of adenovirus-mediated dystrophin minigene transfer in dystrophin-deficient mdx mice.

January 20, 1996Human Gene Therapy

Dystrophin Expression in Muscles of mdx Mice After Adenovirus-Mediated In Vivo Gene Transfer

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Authors

GAGyula AcsádiUniversity of Connecticut HLHanns LochmüllerSemmelweis University ÁJÁgnes JániWayne State University

Discussion

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Implication

Animal study demonstrates that adenoviral dystrophin transfer temporarily protects mdx muscle fibers, indicating host immune responses eliminate transduced cells over time.

Key Points

Evaluate the feasibility, short-term protective efficacy, and long-term persistence of adenovirus-mediated dystrophin minigene transfer in dystrophin-deficient mdx mice.
Injected pure, high-titer adenoviral recombinants encoding a 6.3-kb truncated dystrophin cDNA under RSV or CMV promoters into neonatal, young adult, and aged mdx mice, as well as SCID mice.
Delivered adenoviral vectors systemically via left-ventricular cardiac injection to assess tissue-specific transduction in heart, diaphragm, intercostal, and limb muscles.
Tracked dystrophin expression, muscle fiber morphology, and host CD8+ cytotoxic T-lymphocyte infiltration at 5, 10, 30, and 60 days post-injection.
Transduction at 10 days post-injection yielded high expression of truncated (approximately 200 kD) dystrophin that significantly reduced muscle fiber damage, with higher efficiency in neonates than in adult mice.
Dystrophin-positive fibers substantially declined by 60 days post-injection in neonatally injected muscles and were nearly completely eliminated in adult-injected muscles due to host CD8+ cytotoxic T-cell activity.
Transduced muscle fibers in immunodeficient SCID mice retained early transduction levels up to 30 days post-injection without significant decline.

Cite This Study

Acsádi et al. (1996) studied this question.

synapsesocial.com/papers/6a8407a0833903f64df2edc2 https://doi.org/10.1089/hum.1996.7.2-129

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January 20, 1996Human Gene Therapy

Dystrophin Expression in Muscles of mdx Mice After Adenovirus-Mediated In Vivo Gene Transfer

View Full Paper

Ask AI

Bookmark

Authors

GAGyula AcsádiUniversity of Connecticut HLHanns LochmüllerSemmelweis University ÁJÁgnes JániWayne State University

Discussion

Member takes

Implication

Animal study demonstrates that adenoviral dystrophin transfer temporarily protects mdx muscle fibers, indicating host immune responses eliminate transduced cells over time.

Key Points

Evaluate the feasibility, short-term protective efficacy, and long-term persistence of adenovirus-mediated dystrophin minigene transfer in dystrophin-deficient mdx mice.
Injected pure, high-titer adenoviral recombinants encoding a 6.3-kb truncated dystrophin cDNA under RSV or CMV promoters into neonatal, young adult, and aged mdx mice, as well as SCID mice.
Delivered adenoviral vectors systemically via left-ventricular cardiac injection to assess tissue-specific transduction in heart, diaphragm, intercostal, and limb muscles.
Tracked dystrophin expression, muscle fiber morphology, and host CD8+ cytotoxic T-lymphocyte infiltration at 5, 10, 30, and 60 days post-injection.
Transduction at 10 days post-injection yielded high expression of truncated (approximately 200 kD) dystrophin that significantly reduced muscle fiber damage, with higher efficiency in neonates than in adult mice.
Dystrophin-positive fibers substantially declined by 60 days post-injection in neonatally injected muscles and were nearly completely eliminated in adult-injected muscles due to host CD8+ cytotoxic T-cell activity.
Transduced muscle fibers in immunodeficient SCID mice retained early transduction levels up to 30 days post-injection without significant decline.

Cite This Study

Acsádi et al. (1996) studied this question.

synapsesocial.com/papers/6a8407a0833903f64df2edc2 https://doi.org/10.1089/hum.1996.7.2-129

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