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Ocular Disease and Gene Therapy

Categories

While gene therapy was advancing, it became evident that some tissues are more amenable than others to be targeted by gene delivery. Among them is the eye, which offers several advantages. Retinal gene therapy has advanced considerably in the past three decades. Initial efforts have been devoted to comprehensively explore and optimize the transduction abilities of gene delivery vectors, define the appropriate intraocular administration routes and obtain evidence of efficacy in animal models of inherited retinal diseases (IRDs).1

Schematic representation of the retina and pattern of expression of retinal genes. Genes for which gene therapy approaches are in clinical testing are indicated in bold. RPE: retinal pigment epithelium.

Fig. 1: Schematic representation of the retina and pattern of expression of retinal genes. Genes for which gene therapy approaches are in clinical testing are indicated in bold. RPE: retinal pigment epithelium.1

Inherited Diseases of the Eye

Inherited retinal dystrophies (IRDs) include a number of rare disorders caused by genetic defects. These disorders lead to progressive retinal degeneration. IRDs are well suited for gene therapy as the underlying mutations are well understood. In addition delivery of the therapeutic via AAVs or LVs has proven to work. The development of ocular gene therapy over the prior two decades has mainly targeted hereditary retinal diseases because the genetics of many of these diseases have been well characterized and treatment options for these rare diseases are limited.

The most common IRDs are:3

Leber congenital amaurosis (LCA)

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationELISA, IF, IHC
Validations
  • collections(3)
Cat. No.ABIN334371
Quantity100 μg
ReactivityHuman, Mouse
ClonalityPolyclonal
Clone
ApplicationELISA, ICC, IF, WB
Validations
  • collections(2)
Cat. No.ABIN6257056
Quantity100 μL
ReactivityRat
ClonalityPolyclonal
Clone
ApplicationELISA, WB
Validations
  • collections(2)
Cat. No.ABIN238680
Quantity100 μg
Reactivity
ClonalityMonoclonal
Clone401-8B11-3D9
ApplicationICC, IHC, IHC (fro), WB
Validations
  • (5)
  • collections(2)
Cat. No.ABIN534120
Quantity200 μL

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Choroideremia

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityAvian, Cat, Chicken, Cow, Dog, Donkey, Goat, Guinea Pig, Hamster, Horse, Human, Monkey, Mouse, Pig, Rabbit, Rat, Sheep
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN1440017
Quantity600 μg
ReactivityHorse, Human
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • (1)
  • collections(1)
Cat. No.ABIN2792245
Quantity100 μL
ReactivityHuman
ClonalityMonoclonal
Clone6D7
ApplicationIF, IHC (p), ELISA, WB
Validations
  • (3)
  • collections(6)
Cat. No.ABIN562836
Quantity100 μg
ReactivityHuman
ClonalityMonoclonal
Clone6D7
ApplicationEIA, IF, IHC (p), WB
Validations
  • collections(4)
Cat. No.ABIN781838
Quantity50 μg
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationIHC, WB
Validations
  • collections(8)
Cat. No.ABIN1513519
Quantity100 μg

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Leber's hereditary optic neuropathy (LHON)

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationELISA, WB
Validations
  • collections(1)
Cat. No.ABIN561869
Quantity50 μL
ReactivityMouse
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN6144111
Quantity100 μL
ReactivityRat
ClonalityPolyclonal
Clone
ApplicationELISA, WB
Validations
  • collections(2)
Cat. No.ABIN238680
Quantity100 μg
Reactivity
ClonalityMonoclonal
Clone401-8B11-3D9
ApplicationICC, IHC, IHC (fro), WB
Validations
  • (5)
  • collections(2)
Cat. No.ABIN534120
Quantity200 μL

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Retinitis pigmentosa

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman
ClonalityMonoclonal
CloneMERTK-3015
ApplicationIHC, StM
Validations
  • collections(4)
Cat. No.ABIN6939061
Quantity100 μg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationFACS, IF, IHC, ELISA, WB
Validations
  • collections(7)
Cat. No.ABIN3030229
Quantity0.4 mL
ReactivityCow, Dog, Guinea Pig, Horse, Human, Monkey, Rabbit
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN6748131
Quantity100 μL
ReactivityCow, Dog, Guinea Pig, Horse, Human, Mouse, Rabbit, Rat
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN2789553
Quantity100 μL
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN6146974
Quantity100 μL
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationEIA, WB
Validations
  • collections(2)
Cat. No.ABIN954582
Quantity0.4 mL

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Achromatopsia

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityMouse, Rat
ClonalityPolyclonal
Clone
ApplicationIHC
Validations
  • collections(1)
Cat. No.ABIN1742526
Quantity50 μg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN1679299
Quantity100 μg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationELISA, IHC
Validations
  • collections(1)
Cat. No.ABIN6391408
Quantity100 μg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationEIA, WB
Validations
  • collections(1)
Cat. No.ABIN951585
Quantity0.4 mL

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X-linked retinoschisis (XLRS)

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN519922
Quantity50 μg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationICC, IHC, WB
Validations
  • collections(2)
Cat. No.ABIN1870451
Quantity100 μL

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Usher type 1B syndrome

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationELISA, IF, IHC
Validations
  • collections(2)
  • (1)
Cat. No.ABIN2435036
Quantity200 μL
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationWB
Validations
  • collections(1)
Cat. No.ABIN1680949
Quantity100 μg

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Stargardt's disease

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationELISA, WB
Validations
  • (3)
  • collections(2)
Cat. No.ABIN343052
Quantity100 μg
ReactivityCow
ClonalityMonoclonal
Clone3F4
ApplicationIHC, WB
Validations
  • collections(1)
Cat. No.ABIN361347
Quantity100 μL

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ABCA4 Antibody cited in Stargardt Disease Gene Therapy Article

McClements et al. investigate use of dual vector approach for AAV Gene Therapy of Stargardt disease. anti-ABCA4 antibody (ABIN343052) cited in Human Gene Therapy Vol. 30, No. 5. (2019)

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Non-hereditary Diseases of the Eye

The achievements in clinical trials investigating gene therapy for inherited ocular diseases have prompted the research community to use gene therapy to manage some of the most common nonhereditary ocular diseases, particularly diseases for which the current treatments are effective but are not sustainable in the long term, such as glaucoma, neovascular age-related macular degeneration (AMD), and diabetic retinopathy. Although these diseases involve multiple genes and are more complex and heterogeneous, progress has been made towards treatments in humans.

The most common non-hereditary diseases are:3

Open-angle glaucoma

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationFACS, IF (p), IHC (p), WB
Validations
  • (2)
  • collections(4)
Cat. No.ABIN730153
Quantity100 μL
ReactivityHuman, Rat
ClonalityMonoclonal
Clone5G3B5
ApplicationELISA, FACS, ICC, IHC, WB
Validations
  • collections(5)
Cat. No.ABIN5542567
Quantity0.1 mg
ReactivityHuman, Rat
ClonalityPolyclonal
Clone
ApplicationIHC (p), WB
Validations
  • collections(3)
Cat. No.ABIN2857031
Quantity100 μL

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Age-related macular degeneration (AMD)

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationIHC, IHC (p), WB
Validations
  • collections(1)
Cat. No.ABIN2616159
Quantity0.1 mg
ReactivityHuman
ClonalityMonoclonal
Clone8D2B8
ApplicationICC, FACS, IHC, ELISA, WB
Validations
  • (2)
  • collections(8)
Cat. No.ABIN1098134
Quantity0.1 mg
ReactivityHuman
ClonalityMonoclonal
Clone8D2B8
ApplicationELISA, FACS, IF, IHC, WB
Validations
  • collections(8)
Cat. No.ABIN1491388
Quantity100 μg
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationIF (p), IHC (p), WB
Validations
  • (3)
  • collections(2)
Cat. No.ABIN669951
Quantity100 μL
ReactivityHuman
ClonalityMonoclonal
Clone1837-46
ApplicationIA, IHC (fro), FACS, IHC (p), WB
Validations
  • (2)
Cat. No.ABIN2192047
Quantity100 μg
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationEIA, IHC (p), WB
Validations
  • collections(2)
Cat. No.ABIN500642
Quantity0.1 mg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationFACS, IF (p), IHC (p), WB
Validations
  • (14)
  • collections(6)
Cat. No.ABIN727205
Quantity100 μL
ReactivityHuman, Mouse
ClonalityPolyclonal
Clone
ApplicationIHC (p), WB
Validations
  • (6)
  • collections(3)
Cat. No.ABIN2855448
Quantity100 μL

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Diabetic Macular Edema (DME)

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman, Rat
ClonalityMonoclonal
Clone5G3B5
ApplicationELISA, FACS, ICC, IHC, WB
Validations
  • collections(5)
Cat. No.ABIN5542567
Quantity0.1 mg
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationEIA, IHC (p), WB
Validations
  • collections(2)
Cat. No.ABIN500642
Quantity0.1 mg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationFACS, IF (p), IHC (p), WB
Validations
  • (14)
  • collections(6)
Cat. No.ABIN727205
Quantity100 μL

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Glaucoma

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman, Rat
ClonalityMonoclonal
Clone5G3B5
ApplicationELISA, FACS, ICC, IHC, WB
Validations
  • collections(5)
Cat. No.ABIN5542567
Quantity0.1 mg
ReactivityHuman, Rat
ClonalityPolyclonal
Clone
ApplicationIHC (p), WB
Validations
  • collections(3)
Cat. No.ABIN2857031
Quantity100 μL
ReactivityHuman, Mouse
ClonalityPolyclonal
Clone
ApplicationELISA, ICC, IF, IHC, WB
Validations
  • collections(4)
Cat. No.ABIN6259810
Quantity100 μL

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Diabetic Retinopathy

Product
Reactivity
Clonality
Clone
Application
Validations
Cat. No.
Quantity
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationIHC, IHC (p), WB
Validations
  • collections(1)
Cat. No.ABIN2616159
Quantity0.1 mg
ReactivityHuman, Mouse, Pig, Rat
ClonalityPolyclonal
Clone
ApplicationFACS, IF (p), IHC (p), WB
Validations
  • (1)
  • collections(5)
Cat. No.ABIN730798
Quantity100 μL
ReactivityHuman
ClonalityMonoclonal
Clone8D2B8
ApplicationICC, FACS, IHC, ELISA, WB
Validations
  • (2)
  • collections(8)
Cat. No.ABIN1098134
Quantity0.1 mg
ReactivityHuman
ClonalityMonoclonal
Clone8D2B8
ApplicationELISA, FACS, IF, IHC, WB
Validations
  • collections(8)
Cat. No.ABIN1491388
Quantity100 μg
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationIF (p), IHC (p), WB
Validations
  • (3)
  • collections(2)
Cat. No.ABIN669951
Quantity100 μL
ReactivityHuman
ClonalityMonoclonal
Clone1837-46
ApplicationIA, IHC (fro), FACS, IHC (p), WB
Validations
  • (2)
Cat. No.ABIN2192047
Quantity100 μg
ReactivityHuman, Mouse, Rat
ClonalityPolyclonal
Clone
ApplicationEIA, IHC (p), WB
Validations
  • collections(2)
Cat. No.ABIN500642
Quantity0.1 mg
ReactivityHuman
ClonalityPolyclonal
Clone
ApplicationFACS, IF (p), IHC (p), WB
Validations
  • (14)
  • collections(6)
Cat. No.ABIN727205
Quantity100 μL
ReactivityHuman, Mouse
ClonalityPolyclonal
Clone
ApplicationIHC (p), WB
Validations
  • (6)
  • collections(3)
Cat. No.ABIN2855448
Quantity100 μL

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References

  • (1) Ivana Trapani, Alberto Auricchio, Has retinal gene therapy come of age? From bench to bedside and back to bench, Human Molecular Genetics, Volume 28, Issue R1, October 2019, Pages R108–R118, https://doi.org/10.1093/hmg/ddz130
  • (2) Kathleen Gordon, Amy Del Medico, Ian Sander, Arvind Kumar, Bashar Hamad. Gene therapies in ophthalmic disease. Nature Reviews Drug Discovery 18, 415-416 (2019).
  • (3) https://academic.oup.com/view-large/164513896
  • (4) Jia Hui Lee, Jiang-Hui Wang, Jinying Chen, Fan Li, Thomas L. Edwards, Alex W. Hewitt, Guei-Sheung Liu, Gene therapy for visual loss: Opportunities and concerns, Progress in Retinal and Eye Research, Volume 68, 2019, Pages 31-53
  • (5) Jiang-Hui Wang, Damien Ling, Leilei Tu, Peter van Wijngaarden, Gregory J. Dusting, Guei-Sheung Liu, Gene therapy for diabetic retinopathy: Are we ready to make the leap from bench to bedside?, Pharmacology & Therapeutics, Volume 173, 2017, Pages 1-18
  • (6) Jiang-Hui Wang1 & Georgina Eloise Roberts 1 & Guei-Sheung Liu. Updates on Gene Therapy for Diabetic Retinopathy. Current Diabetes Reports (2020) 20: 22.
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