2021
DOI: 10.1038/d41573-021-00017-7
|View full text |Cite
|
Sign up to set email alerts
|

The clinical landscape for AAV gene therapies

Help me understand this report

Search citation statements

Order By: Relevance

Paper Sections

Select...
3
2

Citation Types

1
306
0
3

Year Published

2021
2021
2024
2024

Publication Types

Select...
4
3
2

Relationship

0
9

Authors

Journals

citations
Cited by 372 publications
(310 citation statements)
references
References 0 publications
1
306
0
3
Order By: Relevance
“…Parents were both excited about the potential positive impact of a one-time disease-modifying therapy and cautious of the potential long-term implications. While clinical experience with AAVmediated gene therapy is increasing, with nearly 150 clinical trials approved by regulatory agencies from different countries -94 of which have been completed and 51 for which the efficacy endpoint was reached [63] -data on long-term effects are still relatively few. More so than ever, the informed consent process for trials of such therapies must provide education with clear, balanced information that enables parents to make well-informed decisions.…”
Section: Discussionmentioning
confidence: 99%
“…Parents were both excited about the potential positive impact of a one-time disease-modifying therapy and cautious of the potential long-term implications. While clinical experience with AAVmediated gene therapy is increasing, with nearly 150 clinical trials approved by regulatory agencies from different countries -94 of which have been completed and 51 for which the efficacy endpoint was reached [63] -data on long-term effects are still relatively few. More so than ever, the informed consent process for trials of such therapies must provide education with clear, balanced information that enables parents to make well-informed decisions.…”
Section: Discussionmentioning
confidence: 99%
“…Currently, AAV vectors have been proven to provide required specificity, efficiency, and safety in basic science and clinical applications (Kuzmin et al, 2021; Naso, Tomkowicz, Perry, & Strohl, 2017; Young, Searle, Onion, & Mautner, 2006). The initial development of the AAV-BR1 virus specific for ECs in the CNS (Korbelin et al, 2016) opened a vast field of opportunities for unmet basic and clinical applications.…”
Section: Discussionmentioning
confidence: 99%
“…As an extension of the CNS, the retina provides a good model for neuro-vascular interactions and has high unmet translational needs for targeting ECs. rAAV have been shown to have high target specificity and have been approved by the FDA for clinical use (Kuzmin et al, 2021). Multiple serotypes have been shown to transduce neurons, glia, and RPE in the retina after subretinal, intravitreal and intravenous administration (Byrne, Lin, Lee, Schaffer, & Flannery, 2015; Hickey et al, 2017).…”
Section: Introductionmentioning
confidence: 99%
“…AAV could become the vector of choice for many gene therapy applications due to its simple structure, unique biology, and a lack of known disease associations ( Naso et al, 2017 ; Rabinowitz et al, 2019 ). The number of clinical trials of AAV-based gene therapies initiated annually increased by more than four times from 2014 to 2017 ( Kuzmin et al, 2021 ). Clinical data from more than 3,000 patients over more than 20years suggest that AAV-based gene therapy is safe and efficient.…”
Section: Introductionmentioning
confidence: 99%