High-Efficacy Gene Therapy For Osteoarthritis

Larta Institute
posted on 10/10/2007

• request more info 
•   
•  LICENSE ONLINE

Technology Description:
UCSD researchers developed a novel approach to protect joint cartilage from degradation during osteoarthritis. Based on new insights into the pathways involved in cartilage degradation in osteoarthritis, the researcher has demonstrated that expressing a key molecule has strong chondroprotective effects in an animal model with surgically induced joint instability. A potentially therapeutic gene has been introduced into the affected knees of the animal by using a high-efficiency non-viral gene delivery method. Weeks after the treatment, there was no sign of degeneration of cartilage in the treated knees and no lesions on the cartilage surface were observed. Furthermore, the cartilage was completely protected from loss of proteoglycans, and chondrocytes showed no sign of disorganization but remained in typical columnar arrays.

UC Santa Barbara researchers have developed a novel method for efficient and simple synthesis of a new multivalent cationic lipid with a double-branched headgroup structure for gene delivery applications.

Combined, these two technologies provide a complete solution for osteoarthritis, with attractive effectiveness shown in animal models

Technology Background:
Osteoarthritis is the most common form of arthritis and affects millions of people. It is a debilitating, progressive disease of the joints characterized by the loss of cartilage tissues with the underlying mechanism of disease is still unresolved. Current therapeutic approaches to treating osteoarthritis largely focus on pain relief and improving (or at least maintaining) mobility that include the use of analgesics, nonsteroidal anti-inflammatory drugs (NSAIDs), COX-2 inhibitors, corticosteroids and viscosupplementation. The nonpharmacological options include physical therapy, exercise, body-weight management, orthotics and surgery. Nevertheless, these treatments are insufficient and may be associated with unwanted side effects.

• Inhibits disease progression itself rather than just symptoms.
• Can be extended to give cationic lipids of different charge, spacer, or lipid chain length.
• Greatly increases transfection efficiency.
• Minimizes known toxicity effects of cationic lipids.