Clinical studies using stem cells for treatment of retinal diseases: state of the
            art

Cotrim, Carina Costa; Jorge, Rodrigo; Oliveira, Maria Carolina; Pieroni, Fabiano; Messias, André; Siqueira, Rubens Camargo

doi:10.5935/0004-2749.20200037

Cited by 6 publications

(3 citation statements)

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“…In such approaches, stem cells from a wide variety of retinal and non-retinal sources are used, including mesenchymal, neural, and embryonic stem cells (non-retinal origin) and ciliary epithelia–derived stem cells and RPE stem cells (retinal origin) 6 – 8 . Currently, several clinical trials are undergoing to investigate the safety and efficacy profiles of stem cell transplantation in retinal degeneration 9 – 11 .…”

Section: Introductionmentioning

confidence: 99%

Induction of Human Umbilical Mesenchymal Stem Cell Differentiation Into Retinal Pigment Epithelial Cells Using a Transwell-Based Co-culture System

et al. 2022

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There is an increasing interest in generating retinal pigment epithelial (RPE) cells from stem cells for treating degenerative eye diseases. However, whether human umbilical cord mesenchymal stem cells (HUCMSCs) can differentiate into RPE-like cells in a co-culture system has not been fully understood. In this study, induction of HUCMSC differentiation into RPE-like cells was performed by co-culturing HUCMSCs and a human RPE-like cell line (ARPE19) in a transwell system and then analyzed for biomarkers using quantitative reverse transcription polymerase chain reaction (RT-PCR) and immunofluorescence staining technique. Moreover, the functional characterization of induced cells was carried out by examining their phagocytic and neurotrophic factor–secreting activities. Our results showed that mRNA expressions of RPE-specific markers—MITF, OTX2, RPE65, PEDF, PME17, and CRALBP—and protein markers—RPE65, CRALBP, and ZO-1—were significantly increased in HUCMSC-derived RPE-like cells. Functional characteristic studies showed that these induced cells were capable of engulfing photoreceptor outer segments and secreting brain-derived neurotrophic factor (BDNF) and glial-derived neurotrophic factor (GDNF), which are typical functions of RPE-like cells. Overall, the study findings indicate that the morphology and proliferation of HUCMSCs can be maintained in a serum-free medium, and differentiation into RPE-like cells can be induced by simply co-culturing HUCMSCs with ARPE19 cells. Thus, the study provides fundamental information regarding the clinical-scale generation of RPE-like cells from HUCMSCs.

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Section: Introductionmentioning

confidence: 99%

Induction of Human Umbilical Mesenchymal Stem Cell Differentiation Into Retinal Pigment Epithelial Cells Using a Transwell-Based Co-culture System

et al. 2022

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“…STGD is particularly devastating because affected individuals lose childhood central vision, which is necessary for common tasks including reading, schooling, driving, and recognizing faces. Ongoing stem cell-based therapy [ 9 , 10 ] or gene therapy [ 11 ] has paved the way to clinical trials for the treatment of STGD. Therefore, accurate and comprehensive molecular diagnosis is critical as an aid to clinical diagnosis, determining the visual prognosis, offering a basis for novel therapeutic approaches, and is also crucial for prenatal diagnosis.…”

Section: Introductionmentioning

confidence: 99%

Targeted next-generation sequencing identifies ABCA4 mutations in Chinese families with childhood-onset and adult-onset Stargardt disease

Qu¹,

Jin

Zeng³

et al. 2021

Bioscience Reports

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Background: Stargardt disease (STGD) is the most common form of juvenile macular dystrophy associated with progressive central vision loss, and is agenetically and clinically heterogeneous disease. Molecular diagnosis is of great significance in aiding the clinical diagnosis, helping to determine the phenotypic severity and visual prognosis. In this study, we determined the clinical and genetic features of seven childhood-onset and three adult-onset Chinese STGD families. We performed capture next generation sequencing (NGS) of the probands and searched for potentially disease-causing genetic variants in previously identified retinal or macular dystrophy genes.  Methods: In all, 10unrelated Chinese families were enrolled. Panel based NGS was performed to identify potentially disease-causing genetic variants in previously identified retinal or macular dystrophy genes, including the five known STGD genes (ABCA4, PROM1, PRPH2, VMD2 and ELOVL4). Variant analysis, Sanger validation, and segregation tests were utilized to validate the disease-causing mutations inthese families. Results: Using systematic data analysis with an established bioinformatics pipeline and segregation analysis, 17 pathogenic mutations in ABCA4 were identified in the ten STGD families. Four of these mutations were novel: c.371delG, c.681T > G, c.5509C > T and EX37del. Childhood-onset STGD was associated with severe visual loss, generalized retinal dysfunction and was due to more severe variants in ABCA4 than those found in adult-onset disease. Conclusions: We expand the existing spectrum of STGD and reveal the genotype-phenotype relationships of the ABCA4 mutations in Chinese patients. Childhood-onset STGD lies at the severe end of the spectrum of ABCA4-associated retinal phenotypes.

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“…Auxiliary treatments are also available for treating the secondary complications of PR, such as cystoid macular edema and cataracts [1,2]. Several techniques using stem cells are being tested in both experimental models and clinical studies to treat the degenerative retinal diseases [3][4][5][6][7][8][9][10][11].…”

Section: Introductionmentioning

confidence: 99%

Improvement of the visual field after intravitreal use of bone marrow mononuclear fraction containing CD34 + stem cells in patients with retinitis pigmentosa: A case report

Siqueira¹,

Piron-Ruiz²,

Saran³

et al. 2021

J Transl Sci

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Background:The objective of this report is to demonstrate a functional retinal rescue in a patient with retinitis pigmentosa (RP) after the intravitreal use of bone marrow mononuclear fraction containing CD34+ stem cells.Case presentation: This report presents the case of a 38-year-old female patient with RP and macular hole, who underwent treatment with the intravitreal injection of bone marrow mononuclear fraction containing CD34+ stem cells and showed an impressive improvement in the visual field after the procedure.Conclusions: Bone marrow mononuclear fraction containing CD34 + stem cells can lead to the functional improvement of retina via the paracrine effect. Studies with a larger number of cases and longer follow-up times are required for the further analysis of the safety and efficacy of this therapeutic possibility.

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Clinical studies using stem cells for treatment of retinal diseases: state of the art

Cited by 6 publications

References 0 publications

Induction of Human Umbilical Mesenchymal Stem Cell Differentiation Into Retinal Pigment Epithelial Cells Using a Transwell-Based Co-culture System

Induction of Human Umbilical Mesenchymal Stem Cell Differentiation Into Retinal Pigment Epithelial Cells Using a Transwell-Based Co-culture System

Targeted next-generation sequencing identifies ABCA4 mutations in Chinese families with childhood-onset and adult-onset Stargardt disease

Improvement of the visual field after intravitreal use of bone marrow mononuclear fraction containing CD34 + stem cells in patients with retinitis pigmentosa: A case report

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