Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …

Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …

The responses of CD8+ T cells to hepatotropic viruses such as hepatitis B range from
dysfunction to differentiation into effector cells, but the mechanisms that underlie these …

Targeted genome editing by artificial nucleases has brought the goal of site-specific
transgene integration and gene correction within the reach of gene therapy. However, its …

Zinc-finger nucleases (ZFNs) allow gene editing in live cells by inducing a targeted DNA
double-strand break (DSB) at a specific genomic locus. However, strategies for …

Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …

Integrative gene transfer methods are limited by variable transgene expression and by the
consequences of random insertional mutagenesis that confound interpretation in gene …

Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by a functional deficit
of type VII collagen protein due to gene defects in the type VII collagen gene (COL7A1) …

Ex vivo gene therapy based on CD34+ hematopoietic stem cells (HSCs) has shown
promising results in clinical trials, but genetic engineering to high levels and in large scale …