# AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

*PRI Rank #3 · Topics and Trends in Most Cited Virus-based gene therapy research Papers, Class of 2026*

*Canonical URL: https://pri.pepkio.com/top-papers/virus-based-gene-therapy-research/2026/rank-3*

| Field | Value |
| --- | --- |
| Rank | #3 |
| 18m citations | 136 |
| Journal | Nature Medicine |
| Year | 2024 |
| DOI | 10.1038/s41591-024-03304-z |
| Corresponding authors | Jerry R. Mendell |
| Institution | Nationwide Children's Hospital, United States |

**Ranking page:** [Topics and Trends in Most Cited Virus-based gene therapy research Papers, Class of 2026](https://pri.pepkio.com/top-papers/virus-based-gene-therapy-research/2026)

**Paper link:** [10.1038/s41591-024-03304-z](https://doi.org/10.1038/s41591-024-03304-z)

## Topics

Duchenne Muscular Dystrophy · DMD gene · Dystrophin · Delandistrogene moxeparvovec · adeno-associated rh74 viral vector · AAV Gene Therapy · micro-dystrophin expression · North Star Ambulatory Assessment · North Star Ambulatory Assessment · Time to Rise · 10-meter Walk/Run · stride velocity 95th centile · 100-meter Walk/Run · 4-step ascent · PROMIS Mobility · Upper Extremity · ambulatory males · Intravenous Administration · Genome Copies Per Kilogram

## Cite this ranking

```
Pepkio Research Index (PRI). Topics and Trends in Most Cited Virus-based gene therapy research Papers, Class of 2026. https://pri.pepkio.com/top-papers/virus-based-gene-therapy-research/2026. Accessed 2026-07-21.

Zheng Su, Tinsley Li, Thematic Shifts in Early-High-Impact Cancer Genomics and Diagnostics Research: A Bibliometric and Semantic Analysis. bioRxiv 2026.07.04.736459; doi: https://doi.org/10.64898/2026.07.04.736459
```