July 30, 2008Human Gene TherapyOpen Access

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

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Why the study?

Does AAV-9 microdystrophin gene therapy improve electrocardiographic abnormalities in a mouse model of Duchenne muscular dystrophy?

Population

Neonatal mdx mice and normal C57BL/10SnJ mice.

Comparison

A single intravenous injection of AAV serotype-9… vs Untreated mdx mice and normal BL10 mice.

Design

Preclinical

Follow-up

4 months

Authors

YYYongping YueUniversity of Missouri YLYi LaiWenzhou Medical University CLChun LongGuiyang Medical University

Discussion

Member takes

Implication

Hypothesis-generating in mdx mice; leaves open translation of AAV-9 microdystrophin to human DMD conduction disease.

Structured PICO

Does AAV-9 microdystrophin gene therapy improve electrocardiographic abnormalities in a mouse model of Duchenne muscular dystrophy?

Population

Neonatal mdx mice (dystrophin-deficient mouse model for Duchenne muscular dystrophy) and normal C57BL/10SnJ (BL10) mice.

Intervention

A single intravenous injection of AAV serotype-9 (AAV-9) microdystrophin vector (1 x 10^12 vector genome particles) at 1 day of age.

Comparator

Untreated mdx mice and normal BL10 mice.

Outcome

Electrocardiographic parameters including heart rate, PR interval, QT interval, QTc, and cardiomyopathy index at 4 months of age.surrogate

AAV-9 microdystrophin gene therapy efficiently transduces the heart and ameliorates early electrocardiographic abnormalities in a mouse model of Duchenne muscular dystrophy.

Limitations

The highly truncated microgene may not completely normalize the cardiomyopathy index.

Cite This Study

Yue et al. (2008) studied this question.

synapsesocial.com/papers/6a81f2beb35cb7638cee3c87 https://doi.org/10.1089/hum.2008.058

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Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

Discussion

Member takes

Implication

Structured PICO

Limitations

Cite This Study

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

Discussion

Member takes

Implication

Structured PICO

Limitations

Cite This Study

Also Consider

Also Consider