The biological activity of AAV vectors for choroideremia gene therapy can be measured by in vitro prenylation of RAB6A
Choroideremia (CHM) is a rare, X-linked recessive retinal dystrophy caused by mutations in the CHM gene. CHM is ubiquitously expressed in human cells and encodes Rab escort protein 1 (REP1). REP1 plays a key role in intracellular trafficking through the prenylation of Rab GTPases, a reaction that ca...
| Main Authors: | , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
Elsevier
2018
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