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- 2018
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Mark
Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis : Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery
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- Contribution to journal › Article
- 2017
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Mark
Rearing light intensity affects inner retinal pathology in a mouse model of x-linked retinoschisis but does not alter gene therapy outcome
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- Contribution to journal › Article
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Mark
Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia
(
- Contribution to journal › Article
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Mark
The clinical phenotype of CNGA3-related achromatopsia : Pretreatment characterization in preparation of a gene replacement therapy trial
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- Contribution to journal › Article
- 2016
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Mark
Regulation and Function of Lentiviral Vector-Mediated TCIRG1 Expression in Osteoclasts from Patients with Infantile Malignant Osteopetrosis : Implications for Gene Therapy
(
- Contribution to journal › Article