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October 1, 2008Human Gene TherapyOpen Access

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

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Authors

WHWilliam W. HauswirthUniversity of North TexasTATomás S. AlemánChildren's Hospital of PhiladelphiaSKShalesh KaushalUniversity of Maryland, Baltimore

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Hauswirth et al. (2008) studied this question.

synapsesocial.com/papers/69ffd2d464548b97a42d7400https://doi.org/10.1089/hum.2008.107
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2 <i>α</i> 1-Antitrypsin (AAT) Vector in AAT-Deficient Adults2006 · 177 citations
  2. 2Clinical Course and Visual Function in a Family With Mutations in the RPE65 Gene2002 · 51 citations
  3. 3Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness2005 · 476 citations
  4. 4Retinal Laminar Architecture in Human Retinitis Pigmentosa Caused by<i>Rhodopsin</i>Gene Mutations2008 · 134 citations
  5. 5Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis1998 · 492 citations