Pluripotent stem cell therapy for retinal diseases

被引：21

作者：

Ahmed, Ishrat ^{[1
]}

Johnston, Robert J., Jr. ^{[2
]}

Singh, Mandeep S. ^{[1
]}

机构：

[1] Johns Hopkins Univ, Sch Med, Wilmer Eye Inst, Baltimore, MD 21205 USA

[2] Johns Hopkins Univ, Dept Biol, Baltimore, MD 21218 USA

来源：

ANNALS OF TRANSLATIONAL MEDICINE | 2021年 / 9卷 / 15期

关键词：

Retinitis pigmentosa (RP); age-related macular degeneration (AMD); embryonic stem cell (ESC); induced pluripotent stem cell (iPSC); retinal organoid; PIGMENT EPITHELIUM; MACULAR DEGENERATION; STARGARDT DISEASE; GENE-THERAPY; AUTOLOGOUS TRANSLOCATION; CHOROID TRANSLOCATION; EFFICIENT GENERATION; CLINICAL-TRIALS; VISUAL FUNCTION; TRANSPLANTATION;

D O I：

10.21037/atm-20-4747

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Pluripotent stem cells (PSCs), which include human embryonic stem cells (hESCs) and induced pluripotent stem cell (iPSC), have been used to study development of disease processes, and as potential therapies in multiple organ systems. In recent years, there has been increasing interest in the use of PSC-based transplantation to treat disorders of the retina in which retinal cells have been functionally damaged or lost through degeneration. The retina, which consists of neuronal tissue, provides an excellent system to test the therapeutic utility of PSC-based transplantation due to its accessibility and the availability of high -resolution imaging technology to evaluate effects. Preclinical trials in animal models of retinal diseases have shown improvement in visual outcomes following subretinal transplantation of PSC-derived photoreceptors or retinal pigment epithelium (RPE) cells. This review focuses on preclinical studies and clinical trials exploring the use of PSCs for retinal diseases. To date, several phase I/II clinical trials in patients with age-related macular degeneration (AMD) and Stargardt disease (STGD1) have demonstrated the safety and feasibility of PSC-derived RPE transplantation. Additional phase I/II clinical trials using PSC-derived RPE or photoreceptor cells for the treatment of AMD, STGD1, and also retinitis pigmentosa (RP) are currently in the pipeline. As this field continues to evolve, additional technologies may enhance PSC-derived cell transplantation through gene-editing of autologous cells, transplantation of more complex cellular structures such as organoids, and monitoring of transplanted cells through novel imaging technologies.

引用

页数：17

共 118 条

[1] Quantitative and Qualitative Evaluation of Photoreceptor Synapses in Developing, Degenerating and Regenerating Retinas [J].

Akiba, Ryutaro ;

Matsuyama, Take ;

Tu, Hung-Ya ;

Hashiguchi, Tomoyo ;

Sho, Junki ;

Yamamoto, Shuichi ;

Takahashi, Masayo ;

Mandai, Michiko .

FRONTIERS IN CELLULAR NEUROSCIENCE, 2019, 13

[2] A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy [J].

Allikmets, R ;

Singh, N ;

Sun, H ;

Shroyer, NE ;

Hutchinson, A ;

Chidambaram, A ;

Gerrard, B ;

Baird, L ;

Stauffer, D ;

Peiffer, A ;

Rattner, A ;

Smallwood, P ;

Li, YX ;

Anderson, KL ;

Lewis, RA ;

Nathans, J ;

Leppert, M ;

Dean, M ;

Lupski, JR .

NATURE GENETICS, 1997, 15 (03) :236-246

[3] Assessment of established techniques to determine developmental and malignant potential of human pluripotent stem cells [J].

Allison, Thomas F. ;

Andrews, Peter W. ;

Avior, Yishai ;

Barbaric, Ivana ;

Benvenisty, Nissim ;

Bock, Christoph ;

Brehm, Jennifer ;

Bruestle, Oliver ;

Damjanov, Ivan ;

Elefanty, Andrew ;

Felkner, Daniel ;

Gokhale, Paul J. ;

Halbritter, Florian ;

Healy, Lyn E. ;

Hu, Tim X. ;

Knowles, Barbara B. ;

Loring, Jeanne F. ;

Ludwig, Tenneille E. ;

Mayberry, Robyn ;

Micallef, Suzanne ;

Mohamed, Jameelah S. ;

Mueller, Franz-Josef ;

Mummery, Christine L. ;

Nakatsuji, Norio ;

Ng, Elizabeth S. ;

Oh, Steve K. W. ;

O'Shea, Orla ;

Pera, Martin F. ;

Reubinoff, Benjamin ;

Robson, Paul ;

Rossant, Janet ;

Schuldt, Bernhard M. ;

Solter, Davor ;

Sourris, Koula ;

Stacey, Glyn ;

Stanley, Edouard G. ;

Suemori, Hirofumi ;

Takahashi, Kazutoshi ;

Yamanaka, Shinya .

NATURE COMMUNICATIONS, 2018, 9

[4] Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice [J].

Allocca, Mariacarmela ;

Doria, Monica ;

Petrillo, Marco ;

Colella, Pasqualina ;

Garcia-Hoyos, Maria ;

Gibbs, Daniel ;

Kim, So Ra ;

Maguire, Albert ;

Rex, Tonia S. ;

Di Vicino, Umberto ;

Cutillo, Luisa ;

Sparrow, Janet R. ;

Williams, David S. ;

Bennett, Jean ;

Auricchio, Alberto .

JOURNAL OF CLINICAL INVESTIGATION, 2008, 118 (05) :1955-1964

[5] Mechanisms of Age-Related Macular Degeneration [J].

Ambati, Jayakrishna ;

Fowler, Benjamin J. .

NEURON, 2012, 75 (01) :26-39

[6] Mutations in REEP6 Cause Autosomal-Recessive Retinitis Pigmentosa [J].

Arno, Gavin ;

Agrawal, Smriti A. ;

Eblimit, Aiden ;

Bellingham, James ;

Xu, Mingchu ;

Wang, Feng ;

Chakarova, Christina ;

Parfitt, David A. ;

Lane, Amelia ;

Burgoyne, Thomas ;

Hull, Sarah ;

Carss, Keren J. ;

Fiorentino, Alessia ;

Hayes, Matthew J. ;

Munro, Peter M. ;

Nicols, Ralph ;

Pontikos, Nikolas ;

Holder, Graham E. ;

Asomugha, Chinwe ;

Raymond, F. Lucy ;

Moore, Anthony T. ;

Plagnol, Vincent ;

Michaelides, Michel ;

Hardcastle, Alison J. ;

Li, Yumei ;

Cukras, Catherine ;

Webster, Andrew R. ;

Cheetham, Michael E. ;

Chen, Rui .

AMERICAN JOURNAL OF HUMAN GENETICS, 2016, 99 (06) :1305-1315

[7] Transplantation of Embryonic and Induced Pluripotent Stem Cell-Derived 3D Retinal Sheets into Retinal Degenerative Mice [J].

Assawachananont, Juthaporn ;

Mandai, Michiko ;

Okamoto, Satoshi ;

Yamada, Chikako ;

Eiraku, Mototsugu ;

Yonemura, Shigenobu ;

Sasai, Yoshiki ;

Takahashi, Masayo .

STEM CELL REPORTS, 2014, 2 (05) :662-674

[8] Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors [J].

Barnea-Cramer, Alona O. ;

Singh, Mandeep ;

Fischer, Dominik ;

De Silva, Samantha ;

McClements, Michelle E. ;

Barnard, Alun R. ;

MacLaren, Robert E. .

MOLECULAR THERAPY, 2020, 28 (03) :830-844

[9] Gene editing prospects for treating inherited retinal diseases [J].

Benati, Daniela ;

Patrizi, Clarissa ;

Recchia, Alessandra .

JOURNAL OF MEDICAL GENETICS, 2020, 57 (07) :437-444

[10] The new paradigm: retinal pigment epithelium cells generated from embryonic or induced pluripotent stem cells [J].

Bharti, Kapil ;

Miller, Sheldon S. ;

Arnheiter, Heinz .

PIGMENT CELL & MELANOMA RESEARCH, 2011, 24 (01) :21-34

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