Protein Carrier Adeno-Associated Virus

被引:0
作者
Hoffmann, Mareike Daniela [1 ]
Sorensen, Ryan James [2 ]
Extross, Ajay [3 ]
He, Yungui [1 ]
Schmidt, Daniel [1 ]
机构
[1] Univ Minnesota, Dept Genet Cell Biol & Dev, Minneapolis, MN 55455 USA
[2] Univ Minnesota, Dept Biochem Mol Biol & Biophys, Minneapolis, MN 55455 USA
[3] Univ Minnesota, Dept Mol Cellular Dev Biol & Genet, Minneapolis, MN 55455 USA
关键词
adeno-associated virus; synthetic virology; protein delivery; nanoparticles; capsid engineering; VIVO GENE-THERAPY; VIRAL VECTORS; DELIVERY; CAPSIDS; IDENTIFICATION; PURIFICATION; STABILITY; EVOLUTION; VARIANTS; PLATFORM;
D O I
10.1021/acsnano.5c01498
中图分类号
O6 [化学];
学科分类号
0703 ;
摘要
Adeno-associated virus (AAV) has emerged as a leading platform for gene therapy, enabling the delivery of therapeutic DNA to target cells. However, the potential of AAV to deliver protein payloads has been unexplored. In this study, we engineered a protein carrier AAV (pcAAV) to package and deliver proteins by inserting binding domains on the interior capsid surface. These binding domains mediate the packaging of specific target proteins through interaction with cognate peptides or protein tags during the capsid assembly process. We demonstrate the packaging of multiple proteins, including green fluorescent protein, Streptococcus pyogenes Cas9, Cre recombinase, and the engineered peroxidase APEX2. Packaging efficiency is modulated by the binding domain insertion site, the viral protein isoform containing the binding domain, and the subcellular localization of the target protein. We show that pcAAV can enter cells and deliver the protein payload and that enzymes retain their activity after packaging. Importantly, this protein packaging capability can be translated to multiple AAV serotypes. Our work establishes AAV as a protein delivery vehicle, significantly expanding the utility of this viral vector for biomedical applications.
引用
收藏
页码:12308 / 12322
页数:15
相关论文
共 74 条
[1]  
Agbandje-McKenna M, 2011, METHODS MOL BIOL, V807, P47, DOI 10.1007/978-1-61779-370-7_3
[2]   Successful liver transduction by re-administration of different adeno-associated virus vector serotypes in mice [J].
Baatartsogt, Nemekhbayar ;
Kashiwakura, Yuji ;
Hiramoto, Takafumi ;
Hayakawa, Morisada ;
Kamoshita, Nobuhiko ;
Ohmori, Tsukasa .
JOURNAL OF GENE MEDICINE, 2023, 25 (08)
[3]   Nanocarriers for oral delivery of biologics: small carriers for big payloads [J].
Cao, Yuxue ;
Rewatkar, Prarthana ;
Wang, Ran ;
Hasnain, Sumaira Z. ;
Popat, Amirali ;
Kumeria, Tushar .
TRENDS IN PHARMACOLOGICAL SCIENCES, 2021, 42 (11) :957-972
[4]   Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems [J].
Chan, Ken Y. ;
Jang, Min J. ;
Yoo, Bryan B. ;
Greenbaum, Alon ;
Ravi, Namita ;
Wu, Wei-Li ;
Sanchez-Guardado, Luis ;
Lois, Carlos ;
Mazmanian, Sarkis K. ;
Deverman, Benjamin E. ;
Gradinaru, Viviana .
NATURE NEUROSCIENCE, 2017, 20 (08) :1172-+
[5]   Endosomal escape: A bottleneck for LNP- mediated therapeutics [J].
Chatterjee, Sushmita ;
Kon, Edo ;
Sharma, Preeti ;
Peer, Dan .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2024, 121 (11)
[6]   In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous [J].
Dalkara, Deniz ;
Byrne, Leah C. ;
Klimczak, Ryan R. ;
Visel, Meike ;
Yin, Lu ;
Merigan, William H. ;
Flannery, John G. ;
Schaffer, David V. .
SCIENCE TRANSLATIONAL MEDICINE, 2013, 5 (189)
[7]   Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain [J].
Deverman, Benjamin E. ;
Pravdo, Piers L. ;
Simpson, Bryan P. ;
Kumar, Sripriya Ravindra ;
Chan, Ken Y. ;
Banerjee, Abhik ;
Wu, Wei-Li ;
Yang, Bin ;
Huber, Nina ;
Pasca, Sergiu P. ;
Gradinaru, Viviana .
NATURE BIOTECHNOLOGY, 2016, 34 (02) :204-+
[8]   Proteomics Analysis of Co-Purifying Cellular Proteins Associated with rAAV Vectors [J].
Dong, Biao ;
Duan, Xunbao ;
Chow, Hoi Yee ;
Chen, Lingxia ;
Lu, Hui ;
Wu, Wenman ;
Hauck, Bernd ;
Wright, Fraser ;
Kapranov, Philipp ;
Xiao, Weidong .
PLOS ONE, 2014, 9 (02)
[9]   Adeno-associated Virus (AAV) Assembly-Activating Protein Is Not an Essential Requirement for Capsid Assembly of AAV Serotypes 4, 5, and 11 [J].
Earley, Lauriel F. ;
Powers, John M. ;
Adachi, Kei ;
Baumgart, Joshua T. ;
Meyer, Nancy L. ;
Xie, Qing ;
Chapman, Michael S. ;
Nakai, Hiroyuki .
JOURNAL OF VIROLOGY, 2017, 91 (03)
[10]   Nanobody-Enhanced Targeting of AAV Gene Therapy Vectors [J].
Eichhoff, Anna Marei ;
Boerner, Kathleen ;
Albrecht, Birte ;
Schaefer, Waldemar ;
Baum, Natalie ;
Haag, Friedrich ;
Koerbelin, Jakob ;
Trepel, Martin ;
Braren, Ingke ;
Grimm, Dirk ;
Adriouch, Sahil ;
Koch-Nolte, Friedrich .
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2019, 15 :211-220