Concepedia

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Albert M. Maguire, Katherine A. High, Alberto Auricchio, J. Fraser Wright, Eric A. Pierce, Francesco Testa, Federico Mingozzi, Jeannette Bennicelli, Gui‐Shuang Ying, Settimio Rossi,

The Lancet · 2009 · 858 citations · 25 references

DOI

Concepts

References

25

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text

, , +17

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

James Bainbridge, Alexander J. Smith, Susie S. Barker et al. · New England Journal of Medicine · 2008 · 1.9K citations · Full text

, , +15

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Salima Hacein‐Bey‐Abina, Alexandrine Garrigue, Gary P. Wang et al. · Journal of Clinical Investigation · 2008 · 1.8K citations · Full text

, , +14

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

Steven J. Howe, Marc R. Mansour, Kerstin Schwarzwaelder et al. · Journal of Clinical Investigation · 2008 · 1.3K citations · Full text

, , +13

PTC124 targets genetic disorders caused by nonsense mutations

Ellen Welch, Elisabeth R. Barton, Jin Zhuo et al. · Nature · 2007 · 1.1K citations

, , +8