Stem Cell Therapy for Genetic and Degenerative Eye Diseases

The beacon of hope for patients with genetic and degenerative eye diseases that used to have limited or no effective treatments has arrived: stem cell therapy. Stem cells are unspecialized cells in the human body that can divide and renew themselves, thus having the potential to develop into different types of specialized cells to perform specific functions. 

The therapy process is one that is rewarding, yet filled with challenges. First, stem cells must be collected, and they can be either autologous (from the patient’s own body) or allogenic (from a donor). Following that, the cells may be altered to adhere to the specific diseases it aims to treat, and from there the cells are administered to the patient through injection into the bloodstream or direct transplantation. After applied, the stem cells replace or repair the diseased or dysfunctional cells of the area as well as protect the remaining healthy cells, restoring normal function for the patient.

In ophthalmology, stem cell therapy is able to target countless diseases ranging from the common glaucoma and diabetic retinopathy to extremely rare diseases such as stargardt or retinitis pigmentosa. Besides embryonic stem cells (ESCs), adult stem cells from bone marrow, umbilical cord, corneal limbus, and retinal lineage are also commonly used for treating these eye diseases although some may go through reprogramming prior to administration. 

As of now, Holoclar stands as the only clinically approved stem cell therapy for the eye, targeted towards restoring vision in patients with corneal damage through the process of transplanting lab-grown limbal stem cells. This treatment has shown long-term safety and great success, with over 80% success in restoring corneal clarity and vision so far, showing great promise for expansion of the field. However, researchers and clinical practitioners are facing many obstacles on their journey. The most prominent issues include the risk factors of immune rejection and tumor formation, as well as ethical issues regarding the farming of embryonic stem cells. Unregulated treatments offered by unlicensed clinics have also surfaced, leading to consequences as severe as permanent vision loss. 

While stem cell therapy is still in a developmental and investigative stage requiring continuous research, technological innovation, and clinical evaluation , it shows promise in becoming a widespread clinical practice, forging a brighter future for patients of the world.

Written by Renee Wang at Incisionary

References:

Mahla R. S. (2016). Stem Cells Applications in Regenerative Medicine and Disease Therapeutics. International journal of cell biology, 2016, 6940283. https://doi.org/10.1155/2016/6940283 

Mayo Clinic Staff. (n.d.). Stem cells: What they are and what they do. Mayo Clinic. Retrieved September 26, 2025, from https://www.mayoclinic.org/tests-procedures/bone-marrow-transplant/in-depth/stem-cells/art-20048117 

Stem cell therapy. (n.d.). The London Clinic. Retrieved September 26, 2025, from https://www.thelondonclinic.co.uk/services/treatments/stem-cell-therapy