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...

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Detalles Bibliográficos
Autores principales: Patrício, Maria I., Barnard, Alun R., Cox, Christopher I., Blue, Clare, MacLaren, Robert E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5918179/
https://www.ncbi.nlm.nih.gov/pubmed/29707603
http://dx.doi.org/10.1016/j.omtm.2018.03.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5918179/
https://www.ncbi.nlm.nih.gov/pubmed/29707603
http://dx.doi.org/10.1016/j.omtm.2018.03.009

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