In vivo tissue-tropism of adeno-associated viral vectors

A Srivastava - Current opinion in virology, 2016 - Elsevier
Highlights•AAV is a non-pathogenic virus, and recombinant AAV vectors have proven to be
highly efficient for gene delivery to a wide variety of cell types, tissue, and organs in small …

Adeno‐associated virus (AAV)-based gene therapy for glioblastoma

X Xu, W Chen, W Zhu, J Chen, B Ma, J Ding… - Cancer Cell …, 2021 - Springer
Glioblastoma (GBM) is the most common and malignant Grade IV primary craniocerebral
tumor caused by glial cell carcinogenesis with an extremely poor median survival of 12–18 …

Widespread central nervous system gene transfer and silencing after systemic delivery of novel AAV-AS vector

SR Choudhury, AF Harris, DJ Cabral, AM Keeler… - Molecular Therapy, 2016 - cell.com
Effective gene delivery to the central nervous system (CNS) is vital for development of novel
gene therapies for neurological diseases. Adeno-associated virus (AAV) vectors have …

Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant‐7m8

H Khabou, M Desrosiers, C Winckler… - Biotechnology and …, 2016 - Wiley Online Library
Recently, we described a modified AAV2 vector—AAV2‐7m8—having a capsid‐displayed
peptide insertion of 10 amino acids with enhanced retinal transduction properties. The …

Structure of neurotropic adeno-associated virus AAVrh. 8

S Halder, K Van Vliet, JK Smith, TTP Duong… - Journal of structural …, 2015 - Elsevier
Adeno-associated virus rhesus isolate 8 (AAVrh. 8) is a leading vector for the treatment of
neurological diseases due to its efficient transduction of neuronal cells and reduced …

A rationally engineered capsid variant of AAV9 for systemic CNS-directed and peripheral tissue-detargeted gene delivery in neonates

D Wang, S Li, DJ Gessler, J Xie, L Zhong, J Li… - … Therapy Methods & …, 2018 - cell.com
Adeno-associated virus (AAV) has provided the gene therapy field with the most powerful in
vivo gene delivery vector to realize safe, efficacious, and sustainable therapeutic gene …

Cryo-electron microscopy reconstruction and stability studies of the wild type and the R432A variant of adeno-associated virus type 2 reveal that capsid structural …

LM Drouin, B Lins, M Janssen, A Bennett… - Journal of …, 2016 - Am Soc Microbiol
The adeno-associated viruses (AAV) are promising therapeutic gene delivery vectors and
better understanding of their capsid assembly and genome packaging mechanism is …

AAV-mediated co-expression of an immunogenic transgene plus PD-L1 enables sustained expression through immunological evasion

TB McMurphy, A Park, PJ Heizer, C Bottenfield… - Scientific Reports, 2024 - nature.com
Adeno-associated virus (AAV) vectors can mediate long-term expression of immunogenic
transgenes in vivo through transduction of tolerogenic cells in the liver. Tissue-targeted AAV …

Compositions for the treatment of disease

S Paul, DT Ward - US Patent 11,326,182, 2022 - Google Patents
The invention provides compositions and methods for the preparation, manufacture and
therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral …

Receptor switching in newly evolved adeno-associated viruses

LP Havlik, A Das, M Mietzsch, DK Oh, J Ark… - Journal of …, 2021 - Am Soc Microbiol
Adeno-associated viruses utilize different glycans and the AAV receptor (AAVR) for cellular
attachment and entry. Directed evolution has yielded new AAV variants; however, structure …