Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo
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Genetic manipulation for inherited neurodegenerative diseases: myth or reality?Recent advances and future prospects in choroideremiaClinical applications of fundus autofluorescence in retinal diseaseWhat Is Next for Retinal Gene Therapy?Biology and therapy of inherited retinal degenerative disease: insights from mouse modelsRetinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.Vector platforms for gene therapy of inherited retinopathies.Gene therapy for choroideremia using an adeno-associated viral (AAV) vector.Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient.Pathogenic mechanisms and the prospect of gene therapy for choroideremia.Retinal Gene Therapy: Current Progress and Future Prospects.Non-viral therapeutic approaches to ocular diseases: An overview and future directionsNovel CHM mutations identified in Chinese families with Choroideremia.Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4-/- mouse and bipolar cells in the rd1 mouse and human retina ex vivo.Genetic analysis and clinical phenotype of two Indian families with X-linked choroideremia.Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human RetinaPrenylation defects in inherited retinal diseases.Stem cells for investigation and treatment of inherited retinal disease.Gene therapies for inherited retinal disorders.Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina.Choroideremia research: Report and perspectives on the second international scientific symposium for choroideremia.Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models.Gene and cell-based therapies for inherited retinal disorders: An update.Multimodal assessment of choroideremia patients defines pre-treatment characteristics.Gene therapy for inherited retinal and optic nerve degenerations.Retinal Gene Therapy for Choroideremia: In Vitro Testing for Gene Augmentation Using an Adeno-Associated Viral (AAV) Vector.Human Retinal Explant Culture for Ex Vivo Validation of AAV Gene Therapy.The Biological Activity of AAV Vectors for Choroideremia Gene Therapy Can Be Measured by In Vitro Prenylation of RAB6A.Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.
P2860
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P2860
Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
Functional expression of Rab e ...... a mice and human cells ex vivo
@en
type
label
Functional expression of Rab e ...... a mice and human cells ex vivo
@en
prefLabel
Functional expression of Rab e ...... a mice and human cells ex vivo
@en
P2093
P2860
P50
P1476
Functional expression of Rab e ...... a mice and human cells ex vivo
@en
P2093
Alun R Barnard
Miguel C Seabra
Nathan J Walker
Tanya Tolmachova
P2860
P2888
P304
P356
10.1007/S00109-013-1006-4
P577
2013-06-12T00:00:00Z