Biomedical Engineering Reference
In-Depth Information
Fig. 1.2 The multiple challenges of cell therapy. Significant progress has been made over the
last years regarding the various aspects of cell therapy (e.g. iPS cells as potential patient-specific
donor cells). However, further multidisciplinary collaborations involving scientists and clinicians
are necessary to advance cell therapies for neurodegenerative disorders
blindness in people over the age of 50. RPE cells are the specific cell type affected in
this disease. Anatomically, the RPE is composed of polarized cells located between
the photoreceptors and the choroid and maintain visual function by serving as a
blood-retina barrier. Since RPE cells exist in a single monolayer that is surgically
accessible, the efficient and controlled generation of these cells from iPS cells could
pave the way to a rational autologous cell replacement therapy in the near future
[
121
]. In 2010, the FDA has approved a clinical trial using human ES cell derived
RPE cells for the treatment of dry AMD [
125
].
1.8 Outlook
Significant advances occurring across scientific disciplines now provide encour-
aging momentum for developing stem cell-based treatments for neurodegenerative
disorders. Personalized therapies for intractable human diseases are no longer a
distant and insurmountable scenario. It is conceivable that combined gene and cell
therapy strategies might be designed and routinely offered to large patient cohorts
with defined disorders that have been characterized with a number of diagnostic
tools including high-throughput whole-genome sequencing, standardized cellular
assays, and high-resolution functional imaging in the clinic. Integrating different
cutting-edge technologies and data sharing will play crucial roles for leveraging
personalized medicine. Comprehensive medical centers for cell therapy should be
established in which collaboration and exchange between scientists and physicians
