A resurgence of interest and investment in the field of gene therapy, driven in large part by
advances in viral vector technology, has recently culminated in United States Food and Drug …

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

The development of gene therapies for the treatment of diseases of the central nervous
system has been hindered by the limited availability of adeno-associated viruses (AAVs) that …

Many disease-causing genes possess functionally equivalent counterparts, which are often
expressed in distinct cell types. An attractive gene therapy approach for inherited disorders …

Delivering and expressing a gene of interest in cells or living animals has become a pivotal
technique in biomedical research and gene therapy. Among viral delivery systems, adeno …

In the past two decades, adeno-associated virus (AAV) vector manufacturing has made
remarkable advancements to meet large-scale production demands for preclinical and …

Direct reprogramming based on genetic factors resembles a promising strategy to replace
lost cells in degenerative diseases such as Parkinson's disease. For this, we developed a …

Astrocytes are particularly promising candidates for reprogramming into neurons, as they
maintain some of the original patterning information from their radial glial ancestors …

Little information is available about how post-transcriptional mechanisms regulate the aging
process. Here, we show that the RNA-binding protein Pumilio2 (PUM2), which is a …

Inflammation is a common feature in neurodegenerative diseases that contributes to
neuronal loss. Previously, we demonstrated that the basal inflammatory tone differed …