ArticleGene therapy2009
Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders.
Article in Gene therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
7 citing papers in PubMed, 20 citations in OpenAlex.
- In Utero Stem Cell Transplantation: Potential Therapeutic Application for Muscle Diseases.Stem cells international · 2017Review
- piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.Nucleic acids research · 2016Article
- Kidney-specific expression of GFP by in-utero delivery of pseudotyped adeno-associated virus 9.Molecular therapy. Methods & clinical development · 2014Article
- Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Therapeutic approaches to muscular dystrophy.Human molecular genetics · 2011Review
- Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene.Gene therapy · 2010Article
- Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
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Authors and funding
4 authors at 3 institutions in 1 country.
Funding
Abstract
One of the major challenges in the treatment of primary muscle disorders, which often affect many muscle groups, is achieving efficient, widespread transgene expression in muscle. In utero gene transfer can potentially address this problem by accomplishing the gene delivery when the tissue mass is small and the immune system is immature. Earlier studies with systemic in utero adeno-associated viral (AAV) vector serotype 1 gene delivery to embryonic day 16 (E-16) pups resulted in high levels of transduction in diaphragm and intercostal muscles, but no detectable transgene expression in limb muscles. Recently, newer AAV serotypes, such as AAV8, have shown widespread and high transgene expression in skeletal muscles and diaphragm by systemic delivery in adult and neonatal mice. We tested AAV8 vector gene delivery by intraperitoneal administration in E-16 mice in utero. Using an AAV8 vector carrying a lacZ reporter gene, we observed high-level transduction of diaphragm and intercostal muscles and more moderate transduction of multiple limb muscles and heart. Our current studies show the potential of AAV8 to achieve widespread muscle transduction in utero and suggest its therapeutic potential for primary muscle disorders.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.