Prenylation of rab6a as a potency assay for choroideremia gene therapy
Choroideremia (CHM) is a rare, X-linked recessive retinal dystrophy caused by mutations in the CHM gene. This gene encodes for Rab escort protein 1 (REP1), which is ubiquitously expressed in human cells and plays a key role in intracellular trafficking through the prenylation of RabGTPases. Deficien...
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| Format: | Conference item |
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Elsevier
2017
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