Gene therapy - are we ready now?

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2022)

AAV transduction and transgene expression engage the host immune system, cellular DNA processing, transcription and translation machineries in ways that have been only cursorily studied in the clinic. Delineating those mechanisms will be key to finding mitigants and solutions to the remaining problems, and including individuals who cannot avail of gene therapy at this time.

Keyphrases

gene therapy
poor prognosis
mental health
circulating tumor
primary care
cell free
single molecule
transcription factor
long non coding rna