Engineering adeno-associated virus vectors for gene therapy

C Li, RJ Samulski - Nature Reviews Genetics, 2020 - nature.com
Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …

[HTML][HTML] The interplay between adeno-associated virus and its helper viruses

AF Meier, C Fraefel, M Seyffert - Viruses, 2020 - mdpi.com
The adeno-associated virus (AAV) is a small, nonpathogenic parvovirus, which depends on
helper factors to replicate. Those helper factors can be provided by coinfecting helper …

Small but increasingly mighty: latest advances in AAV vector research, design, and evolution

D Grimm, H Büning - Human gene therapy, 2017 - liebertpub.com
Recombinant gene delivery vectors derived from naturally occurring or genetically
engineered adeno-associated viruses (AAV) have taken center stage in human gene …

[HTML][HTML] Adeno-associated virus infection and its impact in human health: an overview

TB Sant'Anna, NM Araujo - Virology journal, 2022 - Springer
Discovered as a contaminant of adenovirus stocks in the 1960s, adeno-associated virus
(AAV) is a mono-stranded DNA virus that depends on helper factors to replicate. Even …

Adeno-associated virus monoinfection induces a DNA damage response and DNA repair that contributes to viral DNA replication

K Ning, CA Kuz, F Cheng, Z Feng, Z Yan, J Qiu - Mbio, 2023 - Am Soc Microbiol
Adeno-associated virus (AAV) belongs to the Dependoparvovirus genus of the Parvoviridae
family. AAV replication relies on a helper virus, such as adenovirus (Ad). Co-infection of AAV …

Viral respiratory pathogens and lung injury

N Clementi, S Ghosh, M De Santis… - Clinical microbiology …, 2021 - Am Soc Microbiol
Several viruses target the human respiratory tract, causing different clinical manifestations
spanning from mild upper airway involvement to life-threatening acute respiratory distress …

[HTML][HTML] Viral long non-coding RNA regulates virus life-cycle and pathogenicity

Z Li, J Gao, X Xiang, J Deng, D Gao, X Sheng - Molecular Biology Reports, 2022 - Springer
Viral infection is still a serious global health problem that kills hundreds of thousands of
people annually. Understanding the mechanism by which virus replicates, packages, and …

[HTML][HTML] Virophages of giant viruses: an update at eleven

S Mougari, D Sahmi-Bounsiar, A Levasseur, P Colson… - Viruses, 2019 - mdpi.com
The last decade has been marked by two eminent discoveries that have changed our
perception of the virology field: The discovery of giant viruses and a distinct new class of …

[HTML][HTML] Unravelling the essential elements for recombinant adeno-associated virus (rAAV) production in animal cell-based platforms

D Catalán-Tatjer, K Tzimou, LK Nielsen… - Biotechnology …, 2024 - Elsevier
Recombinant adeno-associated viruses (rAAVs) stand at the forefront of gene therapy
applications, holding immense significance for their safe and efficient gene delivery …

Emerging gene therapies for cystic fibrosis

KM Miah, SC Hyde, DR Gill - Expert review of respiratory medicine, 2019 - Taylor & Francis
Introduction: Cystic fibrosis (CF) remains a life-threatening genetic disease, with few
clinically effective treatment options. Gene therapy and gene editing strategies offer the …