Concepedia

An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque

Rui Xu, Ying Jia, Deborah A. Zygmunt, Megan L. Cramer, Kelly E. Crowe, Guohong Shao, Agatha E. Maki, Haley N. Guggenheim, Benjamin C. Hood, Danielle A. Griffin,

Molecular Therapy — Methods & Clinical Development · 2018 · 16 citations · 48 references

DOIFull text

Open access

Concepts

Abstract

can be accomplished safely using an isolated limb infusion technique, where balloon catheters transiently isolate the limb vasculature, but that intra- and inter-muscle transduction variability is a significant issue.

References

48

Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

Xiao Xiao, Juan Li, R. Jude Samulski · Journal of Virology · 1998 · 1.4K citations · Full text

, , +10

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

Sergei Zolotukhin, Barry J. Byrne, Emily Grace Mason et al. · Gene Therapy · 1999 · 1.3K citations

, , +13

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Christian Hinderer, Nathan Katz, Elizabeth L. Buza et al. · Human Gene Therapy · 2018 · 796 citations · Full text

, , +17

Utrophin-Dystrophin-Deficient Mice as a Model for Duchenne Muscular Dystrophy

Anne E. Deconinck, Jill A. Rafael, Judith A. Skinner et al. · Cell · 1997 · 730 citations · Full text

, , +9

Skeletal and Cardiac Myopathies in Mice Lacking Utrophin and Dystrophin: A Model for Duchenne Muscular Dystrophy

R. Mark Grady, Haibing Teng, Mia C. Nichol et al. · Cell · 1997 · 631 citations · Full text

, , +13