Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors.

Marianna HöselAnke HuberSusanne BohlenJulie LuciforaGiuseppe RonzittiFrancesco PuzzoFlorence BoisgeraultUlrich T HackerWilhelmus Josephus KwantenNora KlötingMatthias BlüherAlexander GluschkoMichael SchrammOlaf UtermöhlenWilhelm BlochFederico MingozziOleg KrutHildegard Büning

Published in: Hepatology (Baltimore, Md.) (2017)

We identified autophagy as a pivotal cell response determining the efficiency of AAVs intracellular processing in hepatocytes and thus the outcome of liver-directed gene therapy using AAV vectors and showed in a proof-of-principle study how this virus-host interaction can be employed to enhance efficacy of this vector system. (Hepatology 2017;66:252-265).

Keyphrases

gene therapy
cell death
endoplasmic reticulum stress
signaling pathway
oxidative stress
sars cov
single cell
cell therapy
stem cells
bone marrow
reactive oxygen species