Načítá se...
Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
INTRODUCTION: Choroideremia is an X-linked inherited retinal degeneration resulting from mutations in the CHM gene, encoding Rab escort protein-1 (REP1), a protein regulating intracellular vesicular transport. Loss-of-function mutations in CHM lead to progressive loss of retinal pigment epithelium (...
Uloženo v:
| Vydáno v: | Expert Opin Orphan Drugs |
|---|---|
| Hlavní autoři: | , , , , , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
2021
|
| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7610829/ https://ncbi.nlm.nih.gov/pubmed/34040899 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/21678707.2021.1882300 |
| Tagy: |
Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!
|