[HTML][HTML] AAV vectors: The Rubik's cube of human gene therapy

A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Gene therapy for the heart lessons learned and future perspectives

A Cannatà, H Ali, G Sinagra, M Giacca - Circulation research, 2020 - Am Heart Assoc
While clinical gene therapy celebrates its first successes, with several products already
approved for clinical use and several hundreds in the final stages of the clinical approval …

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

J El Andari, E Renaud-Gabardos, W Tulalamba… - Science …, 2022 - science.org
Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we …

Next-generation AAV vectors—do not judge a virus (only) by its cover

C Domenger, D Grimm - Human molecular genetics, 2019 - academic.oup.com
Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

[HTML][HTML] Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

J Weinmann, S Weis, J Sippel, W Tulalamba… - Nature …, 2020 - nature.com
Adeno-associated virus (AAV) forms the basis for several commercial gene therapy products
and for countless gene transfer vectors derived from natural or synthetic viral isolates that …

[HTML][HTML] Durability of transgene expression after rAAV gene therapy

M Muhuri, DI Levy, M Schulz, D McCarty, G Gao - Molecular Therapy, 2022 - cell.com
Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the
lives of patients with certain genetic disorders by increasing or restoring function to affected …

Advances in research on pharmacotherapy of sarcopenia

Y Feike, L Zhijie, C Wei - Aging Medicine, 2021 - Wiley Online Library
Sarcopenia is a comprehensive degenerative disease with the progressive loss of skeletal
muscle mass with age, accompanied by the loss of muscle strength and muscle dysfunction …

[HTML][HTML] Towards clinical implementation of adeno-associated virus (AAV) vectors for cancer gene therapy: current status and future perspectives

UT Hacker, M Bentler, D Kaniowska, M Morgan… - Cancers, 2020 - mdpi.com
Adeno-associated virus (AAV) vectors have gained tremendous attention as in vivo delivery
systems in gene therapy for inherited monogenetic diseases. First market approvals …

A small-molecule-responsive riboswitch enables conditional induction of viral vector-mediated gene expression in mice

B Strobel, MJ Düchs, D Blazevic… - ACS Synthetic …, 2020 - ACS Publications
Adeno-associated viral (AAV) vector-mediated gene therapy holds great potential for future
medical applications. However, to facilitate safer and broader applicability and to enable …

Gene Therapeutic Strategies for Peripheral Artery Disease and New Opportunities Provided by Adeno-Associated Virus Vectors

LM Khachigian, RL Varcoe, T Suoranta… - … and Vascular Biology, 2023 - Am Heart Assoc
Peripheral artery disease (PAD) is a vascular disorder caused by occlusive atherosclerosis,
which commonly impairs blood flow to the lower extremities. The prevalence of PAD is …