[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 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
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 …
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 …
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 …
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 …
and for countless gene transfer vectors derived from natural or synthetic viral isolates that …
[HTML][HTML] Durability of transgene expression after rAAV gene therapy
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 …
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 …
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 …
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 …
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 …
which commonly impairs blood flow to the lower extremities. The prevalence of PAD is …