Development of an icIEF assay for monitoring AAV capsid proteins and application to gene therapy products

XZ He, TW Powers, S Huang, Z Liu, H Shi… - … Therapy Methods & …, 2023 - cell.com
Adeno-associated virus (AAV) gene therapy vectors, which contain a DNA transgene
packaged into a protein capsid, have shown tremendous therapeutic potential in recent …

Adeno-associated virus receptor-binding: flexible domains and alternative conformations through Cryo-electron tomography of adeno-associated virus 2 (AAV2) and …

G Hu, MA Silveria, GM Zane, MS Chapman… - Journal of …, 2022 - Am Soc Microbiol
Recombinant forms of adeno-associated virus (rAAV) are vectors of choice in the
development of treatments for a number of genetic dispositions. Greater understanding of …

The large nonstructural protein (NS1) of human bocavirus 1 directly interacts with ku70, which plays an important role in virus replication in human airway epithelia

L Shao, K Ning, J Wang, F Cheng, S Wang… - Journal of …, 2022 - Am Soc Microbiol
ABSTRACT Human bocavirus 1 (HBoV1), an autonomous human parvovirus, causes acute
respiratory tract infections in young children. HBoV1 infects well-differentiated (polarized) …

Human Bocavirus 1 NP1 acts as an ssDNA-binding protein to help AAV2 DNA replication and cooperates with RPA to regulate AAV2 capsid expression

Y Zhao, W Liu, Y Li, J Ma, T Liu, H Cui, Y Deng… - Journal of …, 2024 - Am Soc Microbiol
Adeno-associated virus (AAV) requires co-infection with helper virus for efficient replication.
We previously reported that Human Bocavirus 1 (HBoV1) genes, including NP1, NS2, and …

Deciphering the Origin of DNA Viruses (Replication-Associated Parvo-NS1) That Infect Vertebrates from Invertebrate-Infecting Viruses

PA Desingu, TP Rubeni, NR Sundaresan - Microbiology spectrum, 2023 - Am Soc Microbiol
ABSTRACT DNA replication is a standard and essential function among DNA viruses;
however, this functional domain's common ancestor, origin, and evolutionary path in …

Ex vivo gene therapy for β-hemoglobinpathies and metabolic disorders

M Laurent - 2023 - theses.hal.science
Monogenic disorder are inherited mutation (s) in a single gene that result in abnormal
protein synthesis impairing normal cellular homeostasis and organ function that reduces …