Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

With rapid advances in understanding molecular pathogenesis of human diseases in the era
of genome sciences and systems biology, it is anticipated that increasing numbers of …

Herpesviruses infect the majority of the human population and can cause significant
morbidity and mortality. Herpes simplex virus (HSV) type 1 causes cold sores and herpes …

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 …

Osteoarthritis (OA) is a major cause of disability and morbidity in the aging population. Joint
injury leads to cartilage damage, a known determinant for subsequent development of …

Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease (MND) with no cure.
Recent advances in gene therapy open a new perspective to treat this disorder—particularly …

The development of biological methods over the past decade has stimulated great interest in
the possibility to regenerate human tissues. Advances in stem cell research, gene therapy …

In 2001, the first large animal was successfully treated with a gene therapy that restored its
vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness …

Lysosomal storage disorders (LSDs) are a group of monogenic diseases characterized by
progressive accumulation of undegraded substrates into the lysosome, due to mutations in …

Mitochondria are organelles that have pivotal functions in producing the energy necessary
for life and executing the cell death pathway. Targeting drugs and macromolecules to the …