Comparative study of human embryonic stem cells (hESC) and human induced pluripotent stem cells (hiPSC) as a treatment for retinal dystrophies
Riera, Marina (Institut de Microcirurgia Ocular-IMO (Barcelona, Catalunya))
Fontrodona Montals, Laura (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Albert, Silvia (Radboud University Medical Center. Department of Human Genetics)
Ramírez Mora, Diana (Universitat Autònoma de Barcelona. Departament de Cirurgia)
Seriola, Anna (Centro de Medicina Regenerativa de Barcelona)
Salas, Anna (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Muñoz, Yolanda (Centro de Medicina Regenerativa de Barcelona)
Ramos, David (Universidade de Lisboa. CIISA, Faculdade de Medicina Veterinaria)
Villegas-Perez, Maria Paz (Universidad de Murcia. Departamento de Oftalmología, Optometría, Otorrinolaringología y Anatomía Patológica)
Zapata, Miguel Angel (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Raya, Angel (Centro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina)
Ruberte, Jesus (Universitat Autònoma de Barcelona. Departament de Sanitat i d'Anatomia Animals)
Veiga, Anna (Centro de Medicina Regenerativa de Barcelona)
García Arumí, José (Institut de Microcirurgia Ocular-IMO (Barcelona, Catalunya))

Fecha:	2016
Resumen:	Retinal dystrophies (RD) are major causes of familial blindness and are characterized by progressive dysfunction of photoreceptor and/or retinal pigment epithelium (RPE) cells. In this study, we aimed to evaluate and compare the therapeutic effects of two pluripotent stem cell (PSC)-based therapies. We differentiated RPE from human embryonic stem cells (hESCs) or human-induced pluripotent stem cells (hiPSCs) and transplanted them into the subretinal space of the Royal College of Surgeons (RCS) rat. Once differentiated, cells from either source of PSC resembled mature RPE in their morphology and gene expression profile. Following transplantation, both hESC- and hiPSC-derived cells maintained the expression of specific RPE markers, lost their proliferative capacity, established tight junctions, and were able to perform phagocytosis of photoreceptor outer segments. Remarkably, grafted areas showed increased numbers of photoreceptor nuclei and outer segment disk membranes. Regardless of the cell source, human transplants protected retina from cell apoptosis, glial stress and accumulation of autofluorescence, and responded better to light stimuli. Altogether, our results show that hESC- and hiPSC-derived cells survived, migrated, integrated, and functioned as RPE in the RCS rat retina, providing preclinical evidence that either PSC source could be of potential benefit for treating RD.
Derechos:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Lengua:	Anglès
Documento:	Article ; recerca ; Versió publicada
Publicado en:	Molecular Therapy. Methods & Clinical Development, Vol. 3 (03 2016) , p. 16010, ISSN 2329-0501

DOI: 10.1038/mtm.2016.10
PMID: 27006969

12 p, 3.7 MB

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