Search Results - "Toualbi, Lyes"

The Landscape of Non-Viral Gene Augmentation Strategies for Inherited Retinal Diseases by Toualbi, Lyes, Toms, Maria, Moosajee, Mariya

“…Inherited retinal diseases (IRDs) are a heterogeneous group of disorders causing progressive loss of vision, affecting approximately one in 1000 people…”
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Gene Augmentation of CHM Using Non-Viral Episomal Vectors in Models of Choroideremia by Toualbi, Lyes, Toms, Maria, Almeida, Patrick Vingadas, Harbottle, Richard, Moosajee, Mariya

“…Choroideremia (CHM) is an X-linked chorioretinal dystrophy leading to progressive retinal degeneration that results in blindness by late adulthood. It is…”
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AAV-Mediated Gene Delivery to 3D Retinal Organoids Derived from Human Induced Pluripotent Stem Cells by Garita-Hernandez, Marcela, Routet, Fiona, Guibbal, Laure, Khabou, Hanen, Toualbi, Lyes, Riancho, Luisa, Reichman, Sacha, Duebel, Jens, Sahel, Jose-Alain, Goureau, Olivier, Dalkara, Deniz

“…Human induced pluripotent stem cells (hiPSCs) promise a great number of future applications to investigate retinal development, pathophysiology and cell…”
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OP-08 Analysing the transcriptome of choroideremia patient-derived iPSC-RPE by Linkens, Katy, Owen, Nicholas, Toualbi, Lyes, Moosajee, Mariya

“…IntroductionChoroideremia is an X-linked recessive retinal dystrophy, marked by degeneration of the RPE, photoreceptors and choroid. Choroideremia is caused by…”
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Optogenetic Light Sensors in Human Retinal Organoids by Garita-Hernandez, Marcela, Guibbal, Laure, Toualbi, Lyes, Routet, Fiona, Chaffiol, Antoine, Winckler, Celine, Harinquet, Marylin, Robert, Camille, Fouquet, Stephane, Bellow, Sebastien, Sahel, José-Alain, Goureau, Olivier, Duebel, Jens, Dalkara, Deniz

“…Optogenetic technologies paved the way to dissect complex neural circuits and monitor neural activity using light in animals. In retinal disease, optogenetics…”
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Generation of human iPSC line (UCLi013-A) from a patient with microphthalmia and aniridia, carrying a heterozygous missense mutation c.372C>A p.(Asn124Lys) in PAX6 by Harding, Philippa, Lima Cunha, Dulce, Méjécase, Cécile, Eintracht, Jonathan, Toualbi, Lyes, Sarkar, Hajrah, Moosajee, Mariya

“…A human induced pluripotent stem cell (hiPSC) line (UCLi013-A) was generated from fibroblast cells of a 34-year-old donor with multiple ocular conditions…”
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Generation of two human iPSC lines from patients with autosomal dominant retinitis pigmentosa (UCLi014-A) and autosomal recessive Leber congenital amaurosis (UCLi015-A), associated with RDH12 variants by Sarkar, Hajrah, Méjécase, Cécile, Harding, Philippa, Eintracht, Jonathan, Toualbi, Lyes, Cunha, Dulce Lima, Moosajee, Mariya

“…Induced pluripotent stem cell (iPSC) lines were generated from two patients with RDH12 variants. UCLi014-A is from a patient with heterozygous frameshift…”
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Control of Microbial Opsin Expression in Stem Cell Derived Cones for Improved Outcomes in Cell Therapy by Garita-Hernandez, Marcela, Chaffiol, Antoine, Guibbal, Laure, Routet, Fiona, Khabou, Hanen, Riancho, Luisa, Toualbi, Lyes, Picaud, Serge, Sahel, José-Alain, Goureau, Olivier, Duebel, Jens, Dalkara, Deniz

“…Human-induced pluripotent stem cell (hiPSC) derived organoids have become increasingly used systems allowing 3D-modeling of human organ development, and…”
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Generation of two human control iPS cell lines (UCLi016-A and UCLi017-A) from healthy donors with no known ocular conditions by Méjécase, Cécile, Harding, Philippa, Sarkar, Hajrah, Eintracht, Jonathan, Lima Cunha, Dulce, Toualbi, Lyes, Moosajee, Mariya

“…Two human induced pluripotent stem cell (hiPSC) lines (UCLi016-A and UCLi017-A) were generated from fibroblast cells of 23- and 34-year-old healthy male donors…”
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USH2A-retinopathy: From genetics to therapeutics by Toualbi, Lyes, Toms, Maria, Moosajee, Mariya

“…Bilallelic variants in the USH2A gene can cause Usher syndrome type 2 and non-syndromic retinitis pigmentosa. In both disorders, the retinal phenotype involves…”
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Successful large gene augmentation of USH2A with non-viral episomal vectors by Toms, Maria, Toualbi, Lyes, Almeida, Patrick V., Harbottle, Richard, Moosajee, Mariya

“…USH2A mutations are a common cause of autosomal recessive retinitis pigmentosa (RP) and Usher syndrome, for which there are currently no approved treatments…”
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Loss of the crumbs cell polarity complex disrupts epigenetic transcriptional control and cell cycle progression in the developing retina by Owen, Nicholas, Toms, Maria, Tian, Yuan, Toualbi, Lyes, Richardson, Rose, Young, Rodrigo, Tracey‐White, Dhani, Dhami, Pawan, Beck, Stephan, Moosajee, Mariya

“…The crumbs cell polarity complex plays a crucial role in apical–basal epithelial polarity, cellular adhesion, and morphogenesis. Homozygous variants in human…”
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Feasibility of Direct Vitrectomy-Sparing Subretinal Injection for Gene Delivery in Large Animals by Stranak, Zbynek, Ardan, Taras, Nemesh, Yaroslav, Toms, Maria, Toualbi, Lyes, Harbottle, Richard, Ellederova, Zdenka, Lytvynchuk, Lyubomyr, Petrovski, Goran, Motlik, Jan, Moosajee, Mariya, Kozak, Igor

“…To assess the safety and feasibility of direct vitrectomy-sparing subretinal injection for gene delivery in a large animal model. The experimental Liběchov…”
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