Stem cell-based therapy for diseases of the retinal pigment epithelium: from bench to bedside
Abstract
Age-related macular degeneration (AMD) represents a leading cause of blindness in the elderly, and Stargardt's macular dystrophy (SMD) is the most common form of juvenile-onset macular degeneration. Dry AMD and SMD share an underlying pathophysiology, namely dysfunction and ultimately loss of the retinal pigment epithelium (RPE), suggesting that RPE transplantation may offer a potential treatment strategy for both patient populations. Stem cells have emerged as a promising source of replacement RPE. During the past 15 years, extraordinary strides have been made in the identification, characterization, and differentiation of stem cells. Recently, this large body of basic science and preclinical research has been translated to patient care with the publication of results from Phase 1/2 trials demonstrating safety of transplantation of human embryonic stem cell (hESC)-derived RPE into patients with AMD and SMD. While significant challenges remain before dry AMD and SMD become treatable diseases, the goal has become more tangible.
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If the surgeon does not observe leakage, it is assumed that all the injected volume is successfully delivered.
“If the surgeon does not observe any leakage, it is assumed that all the injected volume is successfully delivered (18–21).”
From Subretinal therapy: technological solutions to surgical and immunological challenges (2022) · cites this paper as [19]
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- Authors
- Sachdeva, M. M., & Eliott, D.
- Affiliations
- Massachusetts Eye and Ear Infirmary (US)
- Corresponding author
- Sachdeva, Mira M.
- Venue
- Seminars in Ophthalmology
- Publisher
- Informa UK Limited
- Published
- 2016-03-03
- Volume
- 31
- Issue
- 1-2
- Pages
- 25-29
- ISSN
- 0882-0538 (ISSN-L)
- 1744-5205
- OpenAlex
- W2294783182
- Cited by
- 28
- References
- 39
- Citations by year
- 2026: 1 · 2025: 2 · 2024: 2 · 2023: 1 · 2022: 4 · 2021: 3 · 2020: 6 · 2019: 2 · 2018: 2 · 2017: 5
- FWCI
- 0.27
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Seminars in Ophthalmology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Health Sciences › Medicine › Ophthalmology › Retinal Diseases and Treatments
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
- Keywords
- stem cell therapy, retinal pigment epithelium, age-related macular degeneration, RPE transplantation, human embryonic stem cells, stem cell differentiation, clinical trials, dry age-related macular degeneration, macular degeneration
- MeSH terms
- Human Embryonic Stem Cells, Stargardt Disease, Humans, Macular Degeneration, Stem Cell Transplantation, Retinal Pigment Epithelium, Geographic Atrophy
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