Gene therapy progress and prospects: the skin – easily accessible, but still far away
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2d ago
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Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD
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Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
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Phase I/II trial of a dendritic cell vaccine transfected with DNA encoding melan A and gp100 for patients with metastatic melanoma
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1w ago
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The estimated annual financial impact of gene therapy in the United States
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Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases
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Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)
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1w ago
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Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
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3w ago
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A multinational survey of potential participant perspectives on ocular gene therapy
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3w ago
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AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia
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3w ago
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