Dr. Tsang: Stem Cell Treatment Research and Its Possible Applicability to AchromatopsiaPresentation Transcript
Edward S. Harkness Eye Institute Stephen Tsang, MD, PhD [email_address] Greater NY Center
Retinal Cell loss in Achromatopsia
Gene Therapy (Cells need to be present)
Other therapies: Retinal Prosthesis
No Gap 20/200, 24 yo 20/80, 15 yo
20/150, 35 yo
Optically Empty Gap 20/150, 48 yo 20/160, 23 yo
20/100, 21 yo
13/15 patients with disorganization/loss of IS/OS junction.
Diameter of IS/OS junction loss corresponded well with the inner margin of the hyperflourescent ring on FAF.
7/15 patients with optically empty gap in fovea
9/15 patients also exhibited a hyper-reflective and thickened external limiting membrane.
Cartoon from NY Times
Induced Pluripotent Stem (iPS) cells retinal cells the patient ’ s personalized cytokine profile W/O biopsy the RPE.
Retinal Cells from iPS cells: transplantation and it will not be rejected.
The human induced pluripotent stem (iPS) cells have been produced by reprogramming somatic cells (skin fibroblast) with a set of 4 transcription factors. The human iPS-cells show striking similarities in their morphological, gene expression, and functional characteristics to human ES-cells, and seem to have acquired the critical ES-cell characteristics of unlimited growth and potential to differentiate to all cell types of human body. Fibroblasts and iPS Cells. (A) Fibroblasts taken from patient skin. (B) Fibroblasts after treatment with OCT4, SOX2, KLF4, and cMYC. (C) iPS colonies after SB431542 and PD0325901 selection. (D) Detail of iPS colonies pictured in (C). Induced Pluripotent Stem Cells can be derived from a patient skin fibroblasts
Second Sight: Retinal Prosthesis Trial Completes First Phase Of Testing Ft. Lauderdale, Fla., May 8 – Researchers from the Keck School of Medicine of the University of Southern California, its Doheny Retina Institute and Second Sight, LLC, are reporting on the initial results of their groundbreaking, FDA-approved feasibility trial of an intraocular retinal prosthesis that appears to be able to restore some degree of sight to the blind.