In this section
@article{Au:2022:10.3389/fmed.2021.809118,
author = {Au, HKE and Isalan, M and Mielcarek, M},
doi = {10.3389/fmed.2021.809118},
journal = {Frontiers in Medicine},
pages = {1--14},
title = {Gene therapy advances: a meta-analysis of AAV usage in clinical settings},
url = {http://dx.doi.org/10.3389/fmed.2021.809118},
volume = {8},
year = {2022}
}
TY - JOUR
AB - Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to drive long-term transgene expression in gene therapy. While animal studies have shown promising results, the translatability of AAVs into clinical settings has been partly limited due to their restricted gene packaging capacities, off-target transduction, and immunogenicity. In this study, we analysed over two decades of AAV applications, in 136 clinical trials. This meta-analysis aims to provide an up-to-date overview of the use and successes of AAVs in clinical trials, while evaluating the approaches used to address the above challenges. First, this study reveals that the speed of novel AAV development has varied between therapeutic areas, with particular room for improvement in Central Nervous System disorders, where development has been slow. Second, the lack of dose-dependent toxicity and efficacy data indicates that optimal dosing regimes remain elusive. Third, more clinical data on the effectiveness of various immune-modulation strategies and gene editing approaches are required to direct future research and to accelerate the translation of AAV-mediated gene therapy into human applications.
AU - Au,HKE
AU - Isalan,M
AU - Mielcarek,M
DO - 10.3389/fmed.2021.809118
EP - 14
PY - 2022///
SN - 2296-858X
SP - 1
TI - Gene therapy advances: a meta-analysis of AAV usage in clinical settings
T2 - Frontiers in Medicine
UR - http://dx.doi.org/10.3389/fmed.2021.809118
UR - https://www.frontiersin.org/articles/10.3389/fmed.2021.809118/full
VL - 8
ER -
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