Evaluating precision medicine approaches for gene therapy in patient-specific cellular models of Bietti crystalline dystrophy

Li, Y., Yang, R. R., Li, Y. S., Hsu, C. W., Jenny, L. A., Kong, Y., Ruan, M. Z., Sparrow, J. R., & Tsang, S. H. (2024). Evaluating precision medicine approaches for gene therapy in patient-specific cellular models of Bietti crystalline dystrophy. JCI insight, 9(16), e177231. https://doi.org/10.1172/jci.insight.177231

Authors:
Yao Li
Richard R Yang
Yong-Shi Li
Chun-Wei Hsu
Laura A Jenny
Yang Kong
Merry Zc Ruan
Janet R Sparrow
Stephen H Tsang
Affiliated Authors:
Yao Li
Yong-Shi Li
Chun-Wei Hsu
Laura A Jenny
Yang Kong
Merry Zc Ruan
Janet R Sparrow
Stephen H Tsang
Columbia Affiliation:
Subjects:
Molecular Mechanisms of Retinal Degeneration and Regeneration (OpenAlex Topic)
Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated proteins (OpenAlex Topic)
Bioluminescence Imaging and Reporter Systems (OpenAlex Topic)
Precision Medicine (MeSH)
Genetic Therapy (MeSH)
Retinal Pigment Epithelium (MeSH)
Induced Pluripotent Stem Cells (MeSH)
Corneal Dystrophies, Hereditary (MeSH)
Dependovirus (MeSH)
Author Keywords:
gene therapy
genetic diseases
ophthalmology
therapeutics
ips cells
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Publication Type:
Article
Unique ID:
10.1172/jci.insight.177231
PMID:
39171529
DOI:
10.1172/jci.insight.177231
Journal:
JCI Insight
Publication Date:
Data Source:
PubMed
Source Link:
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View OpenAlex Record

Record Created: