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Gene and Cell Therapy for Ophthalmology Disease
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Gene and Cell Therapy for Ophthalmology Disease

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: closed (31 March 2023) | Viewed by 8473

Special Issue Editors

Prof. Dr. Mario R. Romano

E-Mail Website
Guest Editor
Department of Biomedical Sciences, Humanitas University, Milan, Italy
Interests: retina; vitreo-retinal surgery; intraocular tamponades; gene therapy; stem cell therapy
Special Issues, Collections and Topics in MDPI journals
Prof. Dr. Antonio Inforzato

E-Mail Website
Guest Editor
Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, 20090 Milan, Italy
Interests: complement system; retinal inflammation; tissue regeneration; angiogenic mediators
Dr. Marianna Paulis

E-Mail Website
Guest Editor
Institute of Genetic and Biomedical Research, National Research Council, 20138 Milan, Italy
Interests: gene therapy; induced pluripotent stem cell
Dr. Hannah Levis

E-Mail Website
Guest Editor
Department of Eye and Vision Science, University of Liverpool, Liverpool L69 3BX, UK
Interests: regenerative medicine; biomimetic tissue; cell microenviroments

Special Issue Information

Dear Colleagues,

The genomics era brought a breakthrough in research that has revolutionized multiple fields of medicine. However, ophthalmic therapeutic applications are still scant, while there are abundant unmet needs. In this perspective, gene and cell therapy can potentially fill multiple gaps. In particular, embryonic stem cells (ESC) and induced pluripotent stem cells (iPSC) have demonstrated a powerful potential in regenerating tissues which can be crucial in ophthalmological degenerative conditions. Gene therapy is already a reality in a limited number of genetic ophthalmological diseases with astonishing results. In this scenario, the medical community along with basic scientists have the potentials to unveil a new era for ophthalmology.

The Special Issue is focused on gene and cell therapy for ophthalmology disease from bench to bedside. Potential topics include, but are not limited, induced pluripotent stem cells, embryonic stem cells, gene replacement, RNA interference, DNA nanoparticles, gene and cell therapy. Molecular research articles as well as review articles are invited.

Prof. Dr. Mario R. Romano
Prof. Dr. Antonio Inforzato 
Dr. Marianna Paulis
Dr. Hannah Levis
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • gene therapy
  • cell therapy
  • stem cells
  • ESC
  • iPSC
  • regenerative ophthalmology

Published Papers (5 papers)

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Research

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14 pages, 6081 KiB  
Article
Inhibiting miR-195-5p Induces Proliferation of Human Corneal Endothelial Cells
by Mohit Parekh, Tiago Ramos, Stefano Ferrari and Sajjad Ahmad
Int. J. Mol. Sci. 2023, 24(14), 11490; https://doi.org/10.3390/ijms241411490 - 15 Jul 2023
Viewed by 1061
Abstract
Transparency of the human cornea is responsible for clear vision, which is maintained by a monolayer of non-proliferative human corneal endothelial cells (HCEnCs). Dysfunction of these cells can result in irreversible corneal blindness. It is important to identify key factors that limit the [...] Read more.
Transparency of the human cornea is responsible for clear vision, which is maintained by a monolayer of non-proliferative human corneal endothelial cells (HCEnCs). Dysfunction of these cells can result in irreversible corneal blindness. It is important to identify key factors that limit the proliferation of HCEnCs and thus attempt to reverse them. Extracellular vesicles contain cargo which includes microRNAs (miRNAs) that can modulate a cellular function. In non small cell lung cancer, expression of miR-195-5p has been shown to inhibit proliferation; therefore, we aimed to investigate the inhibitory effect of miR-195-5p in inducing the proliferation of HCEnCs. Human corneal endothelial cell line (HCEC-12) and primary HCEnCs were cultured with miR-195-5p scramble, mimic or inhibitor. Corneal tissues from human cadaveric and FECD donors, and from pigs, mice and rabbits, were used for RT-PCR. miR-195-5p showed an abundance value of 11,363.31 a.u. When normalized against HCEnCs from cadaveric donors, FECD tissues showed a significant upregulation of miR-195-5p (p < 0.05) but was significantly downregulated in pig (p < 0.001), mouse (p < 0.01) and rabbit (p < 0.001) CEnCs, which have known proliferative capacity. Proliferation, cell doubling, and wound healing rates were significantly higher when miR-195-5p was inhibited. Inhibiting miR-195-5p showed a significant improvement in viability (HEC staining), decreased cell apoptosis (TdT-dNTP staining) and expression of ZO-1, NA+/K+-ATPase and Ki-67 markers. Expression of miR-195-5p is found in HCEnCs and FECD cells, which restricts the proliferation of these cells. However, inhibiting miR-195-5p can induce the proliferation of HCEnCs, which opens exciting directions for future research in prolonging FECD pathogenesis by increasing the proliferative capacity of HCEnCs using anti-miR therapy in vivo. Full article
(This article belongs to the Special Issue Gene and Cell Therapy for Ophthalmology Disease)
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15 pages, 4094 KiB  
Article
Holoprosencephaly with a Special Form of Anophthalmia Result from Experimental Induction of bmp4, Oversaturating BMP Antagonists in Zebrafish
by Johannes Bulk, Valentyn Kyrychenko, Philipp M. Rensinghoff, Zahra Ghaderi Ardekani and Stephan Heermann
Int. J. Mol. Sci. 2023, 24(9), 8052; https://doi.org/10.3390/ijms24098052 - 29 Apr 2023
Viewed by 1213
Abstract
Vision is likely our most prominent sense and a correct development of the eye is at its basis. Early eye development is tightly connected to the development of the forebrain. A single eye field and the prospective telencephalon are situated within the anterior [...] Read more.
Vision is likely our most prominent sense and a correct development of the eye is at its basis. Early eye development is tightly connected to the development of the forebrain. A single eye field and the prospective telencephalon are situated within the anterior neural plate (ANP). During normal development, both domains are split and consecutively, two optic vesicles and two telencephalic lobes emerge. If this process is hampered, the domains remain condensed at the midline. The resulting developmental disorder is termed holoprosencephaly (HPE). The typical ocular finding associated with intense forms of HPE is cyclopia. However, also anophthalmia and coloboma can be associated with HPE. Here, we report that a correct balance of Bone morphogenetic proteins (BMPs) and their antagonists are important for forebrain and eye field cleavage. Experimental induction of a BMP ligand results in a severe form of HPE showing anophthalmia. We identified a dysmorphic forebrain containing retinal progenitors, which we termed crypt-oculoid. Optic vesicle evagination is impaired due to a loss of rx3 and, consecutively, of cxcr4a. Our data further suggest that the subduction of prospective hypothalamic cells during neurulation and neural keel formation is affected by the induction of a BMP ligand. Full article
(This article belongs to the Special Issue Gene and Cell Therapy for Ophthalmology Disease)
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Review

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13 pages, 2688 KiB  
Review
The Role of Subretinal Injection in Ophthalmic Surgery: Therapeutic Agent Delivery and Other Indications
by Domenico Tripepi, Assad Jalil, Naseer Ally, Matilde Buzzi, George Moussa, Pierre-Raphaël Rothschild, Tommaso Rossi, Mariantonia Ferrara and Mario R. Romano
Int. J. Mol. Sci. 2023, 24(13), 10535; https://doi.org/10.3390/ijms241310535 - 23 Jun 2023
Cited by 2 | Viewed by 1907
Abstract
Subretinal injection is performed in vitreoretinal surgery with two main aims, namely, the subretinal delivery of therapeutic agents and subretinal injection of fluid to induce a controlled and localized macular detachment. The growing interest in this technique is mainly related to its suitability [...] Read more.
Subretinal injection is performed in vitreoretinal surgery with two main aims, namely, the subretinal delivery of therapeutic agents and subretinal injection of fluid to induce a controlled and localized macular detachment. The growing interest in this technique is mainly related to its suitability to deliver gene therapy in direct contact with target tissues. However, subretinal injection has been also used for the surgical management of submacular hemorrhage through the subretinal delivery of tissue plasminogen activator, and for the repair of full-thickness macular holes, in particular refractory ones. In the light of the increasing importance of this maneuver in vitreoretinal surgery as well as of the lack of a standardized surgical approach, we conducted a comprehensive overview on the current indications for subretinal injection, surgical technique with the available variations, and the potential complications. Full article
(This article belongs to the Special Issue Gene and Cell Therapy for Ophthalmology Disease)
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24 pages, 739 KiB  
Review
Genetic Aspects of Age-Related Macular Degeneration and Their Therapeutic Potential
by Elisa Stradiotto, Davide Allegrini, Giovanni Fossati, Raffaele Raimondi, Tania Sorrentino, Domenico Tripepi, Gianmaria Barone, Antonio Inforzato and Mario R. Romano
Int. J. Mol. Sci. 2022, 23(21), 13280; https://doi.org/10.3390/ijms232113280 - 31 Oct 2022
Cited by 4 | Viewed by 2039
Abstract
Age-related macular degeneration (AMD) is a complex and multifactorial disease, resulting from the interaction of environmental and genetic factors. The continuous discovery of associations between genetic polymorphisms and AMD gives reason for the pivotal role attributed to the genetic component to its development. [...] Read more.
Age-related macular degeneration (AMD) is a complex and multifactorial disease, resulting from the interaction of environmental and genetic factors. The continuous discovery of associations between genetic polymorphisms and AMD gives reason for the pivotal role attributed to the genetic component to its development. In that light, genetic tests and polygenic scores have been created to predict the risk of development and response to therapy. Still, none of them have yet been validated. Furthermore, there is no evidence from a clinical trial that the determination of the individual genetic structure can improve treatment outcomes. In this comprehensive review, we summarize the polymorphisms of the main pathogenetic ways involved in AMD development to identify which of them constitutes a potential therapeutic target. As complement overactivation plays a major role, the modulation of targeted complement proteins seems to be a promising therapeutic approach. Herein, we summarize the complement-modulating molecules now undergoing clinical trials, enlightening those in an advanced phase of trial. Gene therapy is a potential innovative one-time treatment, and its relevance is quickly evolving in the field of retinal diseases. We describe the state of the art of gene therapies now undergoing clinical trials both in the field of complement-suppressors and that of anti-VEGF. Full article
(This article belongs to the Special Issue Gene and Cell Therapy for Ophthalmology Disease)
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Other

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19 pages, 1877 KiB  
Perspective
How to Set Up Genetic Counselling for Inherited Macular Dystrophies: Focus on Genetic Characterization
by Raffaele Raimondi, Fabiana D’Esposito, Tania Sorrentino, Panos Tsoutsanis, Francesco Paolo De Rosa, Elisa Stradiotto, Gianmaria Barone, Angelica Rizzato, Davide Allegrini, Ciro Costagliola and Mario R. Romano
Int. J. Mol. Sci. 2023, 24(11), 9722; https://doi.org/10.3390/ijms24119722 - 03 Jun 2023
Viewed by 1522
Abstract
Inherited macular dystrophies refer to a group of degenerative conditions that predominantly affect the macula in the spectrum of inherited retinal dystrophies. Recent trends indicate a clear need for genetic assessment services in tertiary referral hospitals. However, establishing such a service can be [...] Read more.
Inherited macular dystrophies refer to a group of degenerative conditions that predominantly affect the macula in the spectrum of inherited retinal dystrophies. Recent trends indicate a clear need for genetic assessment services in tertiary referral hospitals. However, establishing such a service can be a complex task due to the diverse skills required and multiple professionals involved. This review aims to provide comprehensive guidelines to enhance the genetic characterization of patients and improve counselling efficacy by combining updated literature with our own experiences. Through this review, we hope to contribute to the establishment of state-of-the-art genetic counselling services for inherited macular dystrophies. Full article
(This article belongs to the Special Issue Gene and Cell Therapy for Ophthalmology Disease)
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