Chronic graft-versus-host disease (GVHD) profoundly affects the quality of life of long-term
survivors of allogeneic hematopoietic stem cell transplantation (SCT). The eyes are
frequently involved, and dry eye syndrome is the most common manifestation of ocular
chronic GVHD. We explored the role of heat shock protein 47 (HSP47) in ocular GVHD and
developed a novel antifibrotic topical therapy using vitamin A–coupled liposomes containing
HSP47 small interfering RNA (siRNA) against HSP47 (VA-lip HSP47). In a mouse model of …

Introduction Chronic graft-versus-host disease (GVHD) is characterized by multiorgan
fibrosis and affects the quality of life of transplant survivors. Ocular manifestations are found
in 40-60% of GVHD patients, and the most common symptom is dry eye syndrome
associated with lacrimal gland fibrosis. We previously demonstrated heat shock protein 47
(HSP47), a collagen specific molecular chaperone, plays a critical role in excessive collagen
synthesis in chronic GVHD, and systemic administration of vitamin A-coupled liposomes …