Research Topics
Genomes and Genes | Aravind AsokanSummaryAffiliation: University of North Carolina Country: USA Publications
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Detail Information
Publications
Reengineered AAV vectors: old dog, new tricksAravind Asokan
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
Discov Med 9:399-403. 2010..Recent approaches driven by the availability of structural information have yielded a new generation of reengineered AAV vectors...
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscleAravind Asokan
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA
Nat Biotechnol 28:79-82. 2010..These features of AAV2i8 suggest that it is well suited to translational studies in gene therapy of musculoskeletal disorders...
Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivoChengwen Li
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
J Virol 83:6817-24. 2009..Notably, this model system can be exploited to study the kinetics of capsid presentation from different serotypes of AAV and permit the design of novel strategies to block CTL-mediated killing of AAV-transduced cells...
Glycan binding avidity determines the systemic fate of adeno-associated virus type 9Shen Shen
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA
J Virol 86:10408-17. 2012....
Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profilesChengwen Li
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA
J Virol 86:7752-9. 2012..Our results suggest that AAV types can be utilized not only as templates to generate mutants with enhanced transduction efficiency but also as substrates for repeat administration...
Systemic gene transfer to skeletal muscle using reengineered AAV vectorsJana L Phillips
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
Methods Mol Biol 709:141-51. 2011..Methods to evaluate the efficiency of gene transfer to skeletal muscle following intravenous or isolated limb infusion of AAV -vectors in mice are discussed in detail...
Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticlesWuping Li
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
Mol Ther 16:1252-60. 2008..Application of this technology to alternative cell/tissue types using AAV or other viral capsid sequences is likely to yield a new class of biological nanoparticles as vectors for human gene transfer...
Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administrationSteven J Gray
University of North Carolina at Chapel Hill, School of Medicine, Chapel Hill, North Carolina, USA
Curr Protoc Neurosci . 2011..Two detailed methods are provided for viral vector delivery into the rodent brain and spinal cord, and for histological detection of transgene expression of GFP...
Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transferNagesh Pulicherla
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA
Mol Ther 19:1070-8. 2011..Liver-detargeted AAV9 variants described herein are excellent candidates for preclinical evaluation in animal models of cardiac and musculoskeletal disease...
Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypesZhijian Wu
Gene Therapy Center, 7113 Thurston Building, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599-7352, USA
J Virol 80:11393-7. 2006....
Surface loop dynamics in adeno-associated virus capsid assemblyNina DiPrimio
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
J Virol 82:5178-89. 2008....
Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivoChengwen Li
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
J Virol 81:7540-7. 2007..In addition, our data suggest that the mouse model may not mimic the immune response noted in humans and additional modification to AAV vectors may be required for further study in order to elicit a similar cellular immune response...
Production of recombinant adeno-associated viral vectorsVivian W Choi
University of North Carolina, Chapel Hill, North Carolina, USA
Curr Protoc Hum Genet . 2007....
Cellular immune response to cryptic epitopes during therapeutic gene transferChengwen Li
Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599, USA
Proc Natl Acad Sci U S A 106:10770-4. 2009..Such unforeseen epitope generation warrants careful analysis of transgene sequences for ARFs to reduce the potential for adverse events arising from immune responses during clinical gene therapy protocols...
The AAV vector toolkit: poised at the clinical crossroadsAravind Asokan
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
Mol Ther 20:699-708. 2012..This review will provide an overview of preclinical studies with the ever-expanding AAV vector portfolio in large animal models and an update on new lead AAV vector candidates poised for clinical translation...
Production of recombinant adeno-associated viral vectors and use for in vitro and in vivo administrationVivian W Choi
University of North Carolina, Chapel Hill, North Carolina, USA
Curr Protoc Neurosci . 2006..Two detailed methods are provided for viral vector injection into rat brain and for histological detection of transgene expression, including lacZ and GFP...
Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infectionEric D Horowitz
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA
ACS Chem Biol 7:1059-66. 2012..These adverse effects caused by tyrosine oxidation support the notion that interfacial dynamics at the AAV capsid two-fold symmetry axis play a role in externalization of VP N-termini during infection...
Intra- and inter-subunit disulfide bond formation is nonessential in adeno-associated viral capsidsNagesh Pulicherla
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States of America
PLoS ONE 7:e32163. 2012..These studies represent an important step towards understanding the role of inter-subunit interactions that drive AAV capsid assembly...
Terminal N-linked galactose is the primary receptor for adeno-associated virus 9Shen Shen
Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina 27599, USA
J Biol Chem 286:13532-40. 2011..Taken together, the studies described herein provide a molecular basis for low infectivity of AAV9 in vitro and a biochemical strategy to enhance gene transfer by AAV9 vectors in general...
Production of recombinant adeno-associated viral vectors for in vitro and in vivo useVivian W Choi
University of North Carolina, Chapel Hill, North Carolina, USA
Curr Protoc Mol Biol . 2007....
Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entryAravind Asokan
Gene Therapy Center, 7113 Thurston Building, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7352, USA
J Virol 80:8961-9. 2006..The aforementioned results suggest that the internalization of AAV2 in 293 cells might follow a "click-to-fit" mechanism that involves the cooperative binding of heparan sulfate and alpha5beta1 integrin by the AAV2 capsids...
Glycated AAV vectors: chemical redirection of viral tissue tropismEric D Horowitz
Gene Therapy Center, Thurston Bowles Building CB7352, University of North Carolina, Chapel Hill, NC 27599 7352, USA
Bioconjug Chem 22:529-32. 2011..Generation of unnatural amino acid side chains through capsid glycation might serve as an orthogonal strategy to engineer AAV vectors displaying novel tissue tropisms for gene therapy applications...
Adeno-associated virus serotypes: vector toolkit for human gene therapyZhijian Wu
Gene Therapy Center, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
Mol Ther 14:316-27. 2006..Strategies for engineering hybrid AAV vectors derived from AAV serotypes and potential implications of the rapidly expanding AAV vector toolkit are discussed...
Biophysical and ultrastructural characterization of adeno-associated virus capsid uncoating and genome releaseEric D Horowitz
Gene Therapy Center, University of North Carolina, Chapel Hill, NC, USA
J Virol 87:2994-3002. 2013..Our work further defines the biophysical mechanisms underlying adeno-associated virus uncoating and genome release...
