Association of Messenger RNA Level With Phenotype in Patients With Choroideremia: Potential Implications for Gene Therapy Dose

IMPORTANCE: Gene therapy is a promising treatment for choroideremia, an X-linked retinal degeneration. The required minimum level of gene expression to ameliorate degeneration rate is unknown. This can be interrogated by exploring the association between messenger RNA (mRNA) levels and phenotype in...

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Veröffentlicht in:	Archives of ophthalmology (1960) 2020-02, Vol.138 (2), p.128-135
Hauptverfasser:	Fry, Lewis E, Patrício, Maria I, Williams, Jonathan, Aylward, James W, Hewitt, Harriet, Clouston, Penny, Xue, Kanmin, Barnard, Alun R, MacLaren, Robert E
Format:	Artikel
Sprache:	eng
Schlagworte:	Adaptor Proteins, Signal Transducing - genetics Adolescent Child Choroideremia - genetics Choroideremia - therapy Comments Gene expression Gene therapy Genetic Association Studies Genetic Therapy Genotype & phenotype Genotypes Humans Male Online First Original Investigation Patients Phenotype Phenotypes Polymerase chain reaction Retina Retinal degeneration Retrospective Studies RNA, Messenger - analysis Splicing Transcription
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