RESTORATION OF F508-DEL FUNCTION BY TRANSCOMPLEMENTATION: THE PARTNERS MEET IN THE ENDOPLASMIC RETICULUM

BACKGROUND/AIMS: Because of the small size of adeno-associated virus, AAV, the cystic fibrosis conductance regulator, CFTR, cDNA is too large to fit within AAV and must be truncated. We report here on two truncated versions of CFTR, which, when inserted into AAV1 and used to infect airway cells, res...

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Publicado en:Cell Physiol Biochem
Main Authors: Bergbower, Emily Anne Smith, Sabirzhanova, Inna, Boinot, Clément, Guggino, William B., Cebotaru, Liudmila
Formato: Artigo
Idioma:Inglês
Publicado: 2019
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7097968/
https://ncbi.nlm.nih.gov/pubmed/31026390
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.33594/000000089
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