TY - JOUR
T1 - Cell therapy with hiPSC-derived RPE cells and RPCs prevents visual function loss in a rat model of retinal degeneration
AU - Salas Torras, Anna
AU - Duarri, Anna
AU - Fontrodona Montals, Laura
AU - Ramírez Mora, Diana
AU - Badia Pérez, Anna
AU - Isla-Magrané, Helena
AU - Ferreira-de-Souza, Barbara
AU - Zapata, Miguel Angel
AU - Raya, Ángel
AU - García Arumí, José
PY - 2021
Y1 - 2021
N2 - Photoreceptor loss is the principal cause of blindness in retinal degenerative diseases (RDDs). Whereas some therapies exist for early stages of RDDs, no effective treatment is currently available for later stages, and once photoreceptors are lost, the only option to rescue vision is cell transplantation. With the use of the Royal College of Surgeons (RCS) rat model of retinal degeneration, we sought to determine whether combined transplantation of human-induced pluripotent stem cell (hiPSC)-derived retinal precursor cells (RPCs) and retinal pigment epithelial (RPE) cells was superior to RPE or RPC transplantation alone in preserving retinal from degeneration. hiPSC-derived RPCs and RPE cells expressing (GFP) were transplanted into the subretinal space of rats. In vivo monitoring showed that grafted cells survived 12 weeks in the subretinal space, and rats treated with RPE + RPC therapy exhibited better conservation of the outer nuclear layer (ONL) and visual response than RPE-treated or RPC-treated rats. Transplanted RPE cells integrated in the host RPE layer, whereas RPC mostly remained in the subretinal space, although a limited number of cells integrated in the ONL. In conclusion, the combined transplantation of hiPSC-derived RPE and RPCs is a potentially superior therapeutic approach to protect retina from degeneration in RDDs. In this article, Anna Duarri and colleagues aim to develop a novel cell-based therapeutic approach to preserve retinal structure and function in retinal degenerative diseases caused by dysfunctional retinal pigment epithelium (RPE). Giving to the eye support of healthy RPE together with healthy retinal cells protects the retina from degeneration
AB - Photoreceptor loss is the principal cause of blindness in retinal degenerative diseases (RDDs). Whereas some therapies exist for early stages of RDDs, no effective treatment is currently available for later stages, and once photoreceptors are lost, the only option to rescue vision is cell transplantation. With the use of the Royal College of Surgeons (RCS) rat model of retinal degeneration, we sought to determine whether combined transplantation of human-induced pluripotent stem cell (hiPSC)-derived retinal precursor cells (RPCs) and retinal pigment epithelial (RPE) cells was superior to RPE or RPC transplantation alone in preserving retinal from degeneration. hiPSC-derived RPCs and RPE cells expressing (GFP) were transplanted into the subretinal space of rats. In vivo monitoring showed that grafted cells survived 12 weeks in the subretinal space, and rats treated with RPE + RPC therapy exhibited better conservation of the outer nuclear layer (ONL) and visual response than RPE-treated or RPC-treated rats. Transplanted RPE cells integrated in the host RPE layer, whereas RPC mostly remained in the subretinal space, although a limited number of cells integrated in the ONL. In conclusion, the combined transplantation of hiPSC-derived RPE and RPCs is a potentially superior therapeutic approach to protect retina from degeneration in RDDs. In this article, Anna Duarri and colleagues aim to develop a novel cell-based therapeutic approach to preserve retinal structure and function in retinal degenerative diseases caused by dysfunctional retinal pigment epithelium (RPE). Giving to the eye support of healthy RPE together with healthy retinal cells protects the retina from degeneration
KW - Cell therapy
KW - Human-induced pluripotent stem cell
KW - Retinal pigment epithelium
KW - Photoreceptors
KW - Retina degeneration
KW - RCS rat
KW - Regenerative medicine
KW - RPE
KW - IPSC
KW - Subretinal injection
U2 - 10.1016/j.omtm.2021.02.006
DO - 10.1016/j.omtm.2021.02.006
M3 - Article
C2 - 33738324
SN - 2329-0501
VL - 20
SP - 688
EP - 702
JO - Molecular Therapy - Methods and Clinical Development
JF - Molecular Therapy - Methods and Clinical Development
ER -