AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

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AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

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2018 nî lūn-bûn

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2018年の論文

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年學術文章

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2018年學術文章

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AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

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AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

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AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

@en

AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

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AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

@en

AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

@nl

P1476

AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

@en

P2093

C Henrique Alves

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Jan Wijnholds

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P2860

A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Müller cells

Targeted ablation of CRB1 and CRB2 in retinal progenitor cells mimics Leber congenital amaurosis

Effect of gene therapy on visual function in Leber's congenital amaurosis

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

Pals1/Mpp5 is required for correct localization of Crb1 at the subapical region in polarized Muller glia cells

Crumbs homologue 1 is required for maintenance of photoreceptor cell polarization and adhesion during light exposure

Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function

Whole exome sequencing identifies CRB1 defect in an unusual maculopathy phenotype

Loss of CRB2 in the mouse retina mimics human retinitis pigmentosa due to mutations in the CRB1 gene.

Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.

P304

135-151

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P31

scholarly article

P356

10.1007/978-1-4939-7522-8_10

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P407

English

P478

1715

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P50

Jan Wijnholds

C H Alves

P577

2018-01-01T00:00:00Z

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P698

29188511

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