Fig. 1: The main principal of gene replacement therapy using a viral vector.
From: Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
Binding of Adeno-associated virus (AAV)-based vectors to specific membrane receptors (1) induces cellular internalization through endocytosis (2). Once inside the host cells, the viral vectors are released from the endosome (3) and shuttled into the nucleus, where the ssDNA is released (4) and undergoes second-strand synthesis to form double-stranded DNA. Subsequently, the transgene is transcribed into corresponding mRNAs (5) that will be translocated to the cytoplasm to generate therapeutic proteins (6). Created with BioRender.com (Agreement number : YC26MKZXYB).
