Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives

MF Dias, K Joo, JA Kemp, SL Fialho… - Progress in retinal and …, 2018 - Elsevier
Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people
worldwide. It is characterized with progressive loss of rods and cones and causes severe …

Generation of three-dimensional retinal tissue with functional photoreceptors from human iPSCs

X Zhong, C Gutierrez, T Xue, C Hampton… - Nature …, 2014 - nature.com
Many forms of blindness result from the dysfunction or loss of retinal photoreceptors.
Induced pluripotent stem cells (iPSCs) hold great potential for the modelling of these …

Thyroid hormone signaling specifies cone subtypes in human retinal organoids

KC Eldred, SE Hadyniak, KA Hussey, B Brenerman… - Science, 2018 - science.org
INTRODUCTION Cone photoreceptors in the human retina enable daytime, color, and high-
acuity vision. The three subtypes of human cones are defined by the visual pigment that they …

Negligible immunogenicity of terminally differentiated cells derived from induced pluripotent or embryonic stem cells

R Araki, M Uda, Y Hoki, M Sunayama, M Nakamura… - Nature, 2013 - nature.com
The advantages of using induced pluripotent stem cells (iPSCs) instead of embryonic stem
(ES) cells in regenerative medicine centre around circumventing concerns about the ethics …

Emerging therapies for inherited retinal degeneration

HPN Scholl, RW Strauss, MS Singh… - Science translational …, 2016 - science.org
Inherited retinal degenerative diseases, a genetically and phenotypically heterogeneous
group of disorders, affect the function of photoreceptor cells and are among the leading …

Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina

A Gonzalez-Cordero, EL West, RA Pearson… - Nature …, 2013 - nature.com
Irreversible blindness caused by loss of photoreceptors may be amenable to cell therapy.
We previously demonstrated retinal repair and restoration of vision through transplantation …

[HTML][HTML] Stemming retinal regeneration with pluripotent stem cells

ZB **, ML Gao, WL Deng, KC Wu, S Sugita… - Progress in retinal and …, 2019 - Elsevier
Cell replacement therapy is a promising treatment for irreversible retinal cell death in diverse
diseases, such as age-related macular degeneration (AMD), Stargardt's disease, retinitis …

Biology and therapy of inherited retinal degenerative disease: insights from mouse models

S Veleri, CH Lazar, B Chang… - Disease models & …, 2015 - journals.biologists.com
Retinal neurodegeneration associated with the dysfunction or death of photoreceptors is a
major cause of incurable vision loss. Tremendous progress has been made over the last two …

[HTML][HTML] Transplantation of photoreceptors into the degenerative retina: Current state and future perspectives

SJ Gasparini, S Llonch, O Borsch, M Ader - Progress in Retinal and Eye …, 2019 - Elsevier
The mammalian retina displays no intrinsic regenerative capacities, therefore retinal
degenerative diseases such as age-related macular degeneration (AMD) or retinitis …