# Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective

Review · Eric L. Hastie, R. Jude Samulski

- Archive id: doi:10.1089/hum.2015.025
- Published: 2015-03-23
- Venue: Human Gene Therapy
- DOI: https://doi.org/10.1089/hum.2015.025
- PMID: 25807962
- Citations: 282
- Source URL: https://doi.org/10.1089/hum.2015.025
- Provenance: openalex

## Abstract

Fifty years after the discovery of adeno-associated virus (AAV) and more than 30 years after the first gene transfer experiment was conducted, dozens of gene therapy clinical trials are in progress, one vector is approved for use in Europe, and breakthroughs in virus modification and disease modeling are paving the way for a revolution in the treatment of rare diseases, cancer, as well as HIV. This review will provide a historical perspective on the progression of AAV for gene therapy from discovery to the clinic, focusing on contributions from the Samulski lab regarding basic science and cloning of AAV, optimized large-scale production of vectors, preclinical large animal studies and safety data, vector modifications for improved efficacy, and successful clinical applications.

_Abstract reproduced under CC-BY (publisher license, via OpenAlex)._

Topics: [Gene & Cell Therapy](https://space-kitty.com/topics/gene-cell-therapy)

---
Space Kitty Archive · https://space-kitty.com · API: https://space-kitty.com/api/v1 · MCP: https://space-kitty.com/mcp · Premium: $0.20/request via x402 (Base Sepolia (testnet)). Metadata: arXiv (CC0), OpenAlex (CC0), Europe PMC (CC BY/CC0 works), ClinicalTrials.gov.