The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Lipid nanoparticles have attracted significant interests in the last two decades, and have
achieved tremendous clinical success since the first clinical approval of Doxil in 1995. At the …

There have been many ups and downs since the introduction of gene therapy as a
therapeutic modality for diseases. However, the journey of gene therapy has reached a …

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 …

A series of recent discoveries harnessing the adaptive immune system of prokaryotes to
perform targeted genome editing is having a transformative influence across the biological …

Gene therapy is on its way to revolutionize the treatment of both inherited and acquired
diseases, by transferring nucleic acids to correct a disease-causing gene in the target cells …

Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential
for treating and curing genetic disorders. For otherwise incurable indications, including most …

Gene vectors are nucleic acids that carry genetic materials or gene editing devices into cells
to exert the sustained production of therapeutic proteins or to correct erroneous genes of the …