Age related macular degeneration – challenge for future: Pathogenesis and new perspectives for the treatment

Michalska-Małecka, Katarzyna; Kabiesz, Adam; Nowak, Mariusz; Śpiewak, Dorota

doi:10.1016/j.eurger.2014.09.007

Cited by 40 publications

(31 citation statements)

References 87 publications

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“…AMD is the most common cause of severe vision loss in patients over the age of 60 and the major cause of blindness in industrialized countries. 36 , 37 , 38 There are two distinct types of AMD: a slowly progressing dry (atrophic) form and a rapidly developing wet (neovascular) form, in which choroidal blood vessels grow through Bruch’s membrane into the subretinal space. The symptoms are characterized by the degeneration of RPE cells, alterations in Bruch’s membrane, neural retinal ganglion cell degradation, and the death of photoreceptor cells.…”

Section: Introductionmentioning

confidence: 99%

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

Pastor

Johnen

Harmening

et al. 2018

Molecular Therapy - Nucleic Acids

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The anti-angiogenic and neurogenic pigment epithelium-derived factor (PEDF) demonstrated a potency to control choroidal neovascularization in age-related macular degeneration (AMD) patients. The goal of the present study was the development of an efficient and safe technique to integrate, ex vivo, the PEDF gene into retinal pigment epithelial (RPE) cells for later transplantation to the subretinal space of AMD patients to allow continuous PEDF secretion in the vicinity of the affected macula. Because successful gene therapy approaches require efficient gene delivery and stable gene expression, we used the antibiotic-free pFAR4 mini-plasmid vector to deliver the hyperactive Sleeping Beauty transposon system, which mediates transgene integration into the genome of host cells. In an initial study, lipofection-mediated co-transfection of HeLa cells with the SB100X transposase gene and a reporter marker delivered by pFAR4 showed a 2-fold higher level of genetically modified cells than when using the pT2 vectors. Similarly, with the pFAR4 constructs, electroporation-mediated transfection of primary human RPE cells led to 2.4-fold higher secretion of recombinant PEDF protein, which was still maintained 8 months after transfection. Thus, our results show that the pFAR4 plasmid is a superior vector for the delivery and integration of transgenes into eukaryotic cells.

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Section: Introductionmentioning

confidence: 99%

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

Pastor

Johnen

Harmening

et al. 2018

Molecular Therapy - Nucleic Acids

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“…AMD is the principal cause of vision loss among older patients. Advanced AMD is characterized by the occurrence of either geographic atrophy (dry form) or choroidal neovascularization (wet form) [45]. Owing to increasing knowledge of the multiple pathological mechanisms associated with both forms of advanced AMD, mainly characterized by chronic inflammation and oxidative stress, several promising therapies are in various stages of development.…”

Section: Age-related Macular Degenerationmentioning

confidence: 99%

Therapeutic application of antibody fragments in autoimmune diseases: current state and prospects

Fernandes

2018

Drug Discovery Today

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“…CNTF is an effective neurotrophic factor that slows down the loss of photoreceptors. The CNTF implants that used in AMD patient slowed the visual loss rate at 12 months [86,87].…”

Section: Age-related Macular Degeneration (Amd)mentioning

confidence: 99%

Ciliary neurotrophic factor (CNTF) delivery to retina: an overview of current research advancements

Ghasemi

Arezoumand

Motlagh

et al. 2017

Artificial Cells, Nanomedicine, and Biotechnology

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The intraocular administration of the ciliary neurotrophic factor (CNTF) has been found to attenuate the photoreceptor degeneration and preserve retinal functions in the animal research models of the inherited or induced retinal disease. Studies with the aim of CNTF transfer to the posterior segment inside the eye have been directed to determine the best method for its administration. An ideal delivery method would overcome the eye drug elimination mechanisms or barriers and provide the sustained release of the CNTF into retina in the safest fashion with the minimum harm to the quality of life. This review focuses on the present state of CNTF delivery to retina, also provides an overview of available technologies and their challenges.

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Age related macular degeneration – challenge for future: Pathogenesis and new perspectives for the treatment

Cited by 40 publications

References 87 publications

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells

Therapeutic application of antibody fragments in autoimmune diseases: current state and prospects

Ciliary neurotrophic factor (CNTF) delivery to retina: an overview of current research advancements

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