Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that
will drive biological discovery and transform the field of cell and gene therapy. Here, we …

Our ability to edit genomes lags behind our capacity to sequence them, but the growing
understanding of CRISPR biology and its application to genome, epigenome and …

Nucleic acids are major structures detected by the innate immune system. Although
intracellular single-stranded DNA (ssDNA) accumulates during pathogen infection or …

Mucopolysaccharidosis (MPS) IVA is a lysosomal storage disorder caused by mutations in
the GALNS gene that leads to the lysosomal accumulation of keratan sulfate (KS) and …

Therapeutic genome editing of haematopoietic stem cells (HSCs) would provide long-lasting
treatments for multiple diseases. However, the in vivo delivery of genetic medicines to HSCs …

Gene therapy has gained interest over the last decades. Several therapies employing gene
transfer mechanisms have been developed, and, of these, adeno-associated virus (AAV) …

Gene therapy is a technique to correct genetic abnormalities, through introduction of a
functional gene or through direct genome editing. Adeno-associated virus (AAV)-mediated …

Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …

Onasemnogene abeparvovec has been life-changing for children with spinal muscular
atrophy (SMA), signifying the potential and progress occurring in gene-and cell-based …