Evolving AAV-delivered therapeutics towards ultimate cures.
Level 5 - mechanism / opinion, no new human data
Narrative review without systematic search or quantitative data synthesis
PubMed 33594520 · doi:10.1007/s00109-020-02034-2
What was done
The authors reviewed the clinical progress of recombinant adeno-associated virus (AAV) gene therapies, the use of AAV to deliver CRISPR components for somatic gene editing, and the major delivery challenges and toxicities observed in clinical applications.
What was found
The abstract reports no primary quantitative findings or effect estimates. It provides a qualitative summary noting the regulatory approvals of Glybera (EMA, 2012), Luxturna (FDA), and Zolgensma (FDA), alongside ongoing developments in CRISPR-based gene editing and clinical safety concerns.
Why it matters
This review outlines the clinical evolution of AAV vectors from traditional gene replacement to precision genome editing while highlighting persistent translational and toxicity challenges.
Limits
As a narrative review, it contains no primary data, systematic search protocol, or meta-analytic pooling. No specific sample sizes, trial outcomes, or numerical toxicity rates are provided in the abstract.
Cited by
- supports Most regulatory approved gene therapies work by gene addition rather than by gene editing.