Title:Current Progress in Stem Cell-Based Gene Therapy for Articular Cartilage Repair
Volume: 10
Issue: 2
Author(s): Janina Frisch, Jagadeesh K. Venkatesan, Ana Rey-Rico, Henning Madry and Magali Cucchiarini
Affiliation:
Keywords:
Cartilage repair, gene therapy, stem cells.
Abstract: Administration of mesenchymal stem cells (MSCs) that have a reliable potential for chondrogenic
differentiation is a promising approach currently employed to treat articular cartilage lesions (focal defects
and osteoarthritis) in patients as a mean to enhance the poor intrinsic capabilities of this specialized tissue for
self-repair. However, there is still a critical need for improved designs, as reproduction of a native structural
and functional unit in sites of cartilage damage is not occurring upon implantation of such cells. With the
availability of optimized gene transfer systems, gene therapy offers powerful tools to stimulate the chondrogenic process
in MSCs via the effective, safe, and durable delivery of candidate sequences with chondroprotective and/or chondroregenerative
properties, both in vitro and in experimental models of cartilage lesions in vivo. In the present article, we provide
an overview of the current advances in gene- and stem cell-based treatments employed to promote cartilage repair in focal
defects and for osteoarthritis, and discuss the challenges that remain to be addressed for a safe translation of such procedures
into the clinics.