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Ocular Disease and Gene Therapy

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).

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.

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:

Leber congenital amaurosis (LCA)

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human
Clonality Polyclonal
Application ELISA, IF, IHC
Validations
  • (3)
Cat. No. ABIN334371
Quantity 100 μg
Datasheet Datasheet
Reactivity Human, Mouse
Clonality Polyclonal
Application ELISA, ICC, IF, WB
Validations
  • (2)
Cat. No. ABIN6257056
Quantity 100 μL
Datasheet Datasheet
Reactivity Rat
Clonality Polyclonal
Application ELISA, WB
Validations
  • (2)
Cat. No. ABIN238680
Quantity 100 μg
Datasheet Datasheet

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Anti-Human Secondary Antibodies

Unconjugated or with enzymatic conjugates like AP, Biotin, HRP or fluorescent conjugates like the Alexa Fluor family.

Variety of different host, isotypes and binding specifities available.

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Choroideremia

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet

Clonality Polyclonal
Application WB
Validations
  • (1)
Cat. No. ABIN1440017
Quantity 600 μg
Datasheet Datasheet
Reactivity Horse, Human
Clonality Polyclonal
Application WB
Validations
  • (1)
  • (1)
Cat. No. ABIN2792245
Quantity 100 μL
Datasheet Datasheet
Reactivity Human
Clonality Monoclonal
Application IF, IHC (p), ELISA, WB
Validations
  • (3)
  • (6)
Cat. No. ABIN562836
Quantity 100 μg
Datasheet Datasheet

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Leber's hereditary optic neuropathy (LHON)

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Mouse
Clonality Polyclonal
Application WB
Validations
  • (1)
Cat. No. ABIN6144111
Quantity 100 μL
Datasheet Datasheet
Reactivity Human
Clonality Polyclonal
Application ELISA, WB
Validations
  • (1)
Cat. No. ABIN561869
Quantity 50 μL
Datasheet Datasheet
Reactivity Rat
Clonality Polyclonal
Application ELISA, WB
Validations
  • (2)
Cat. No. ABIN238680
Quantity 100 μg
Datasheet Datasheet

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Retinitis pigmentosa

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human
Clonality Monoclonal
Application IHC, IHC (f)
Validations
  • (4)
Cat. No. ABIN6939061
Quantity 100 μg
Datasheet Datasheet
Reactivity Human
Clonality Polyclonal
Application FACS, IF, IHC, ELISA, WB
Validations
  • (7)
Cat. No. ABIN3030229
Quantity 0.4 mL
Datasheet Datasheet
Reactivity Cow, Dog, Guinea Pig, Horse, Human, Monkey, Rabbit
Clonality Polyclonal
Application WB
Validations
  • (1)
Cat. No. ABIN6748131
Quantity 100 μL
Datasheet Datasheet
Reactivity Cow, Dog, Guinea Pig, Horse, Human, Mouse, Rabbit, Rat
Clonality Polyclonal
Application WB
Validations
  • (1)
Cat. No. ABIN2789553
Quantity 100 μL
Datasheet Datasheet
Reactivity Mouse
Clonality Polyclonal
Application IHC, WB
Validations
  • (1)
Cat. No. ABIN350792
Quantity 100 μL
Datasheet Datasheet
Reactivity Mouse
Clonality Polyclonal
Application IHC, WB
Validations
  • (1)
Cat. No. ABIN7271760
Quantity 500 μg
Datasheet Datasheet
Reactivity Human
Clonality Polyclonal
Application EIA, WB
Validations
  • (2)
Cat. No. ABIN954582
Quantity 0.4 mL
Datasheet Datasheet

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Achromatopsia

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Mouse, Rat
Clonality Polyclonal
Application IHC
Validations
  • (1)
Cat. No. ABIN1742526
Quantity 50 μg
Datasheet Datasheet
Reactivity Human
Clonality Polyclonal
Application ELISA, IHC
Validations
  • (1)
Cat. No. ABIN6391408
Quantity 100 μg
Datasheet Datasheet
Reactivity Human
Clonality Polyclonal
Application EIA, WB
Validations
  • (1)
Cat. No. ABIN951585
Quantity 0.4 mL
Datasheet Datasheet

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X-linked retinoschisis (XLRS)

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human
Clonality Polyclonal
Application WB
Validations
  • (1)
Cat. No. ABIN519922
Quantity 50 μg
Datasheet Datasheet

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Usher type 1B syndrome

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human, Mouse
Clonality Polyclonal
Application ELISA, IHC
Validations
  • (2)
  • (1)
Cat. No. ABIN2435036
Quantity 200 μL
Datasheet Datasheet

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Stargardt's disease

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human, Mouse
Clonality Polyclonal
Application ELISA, IF
Validations
  • (3)
  • (4)
Cat. No. ABIN343052
Quantity 100 μg
Datasheet Datasheet
Reactivity Cow
Clonality Monoclonal
Application IHC, WB
Validations
  • (1)
Cat. No. ABIN361347
Quantity 100 μL
Datasheet Datasheet

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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:

Open-angle glaucoma

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human, Mouse, Rat
Clonality Polyclonal
Application FACS, IF (cc), IF (p), IHC (fro), IHC (p), WB
Validations
  • (2)
  • (4)
Cat. No. ABIN730153
Quantity 100 μL
Datasheet Datasheet
Reactivity Human, Rat
Clonality Monoclonal
Application ELISA, FACS, ICC, IHC, WB
Validations
  • (5)
Cat. No. ABIN5542567
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human, Rat
Clonality Polyclonal
Application IHC (p), WB
Validations
  • (3)
Cat. No. ABIN2857031
Quantity 100 μL
Datasheet Datasheet

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Age-related macular degeneration (AMD)

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human
Clonality Polyclonal
Application IHC, IHC (p), WB
Validations
  • (1)
Cat. No. ABIN2616159
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human
Clonality Monoclonal
Application ICC, FACS, IHC, ELISA, WB
Validations
  • (2)
  • (8)
Cat. No. ABIN1098134
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human
Clonality Monoclonal
Application ELISA, FACS, IF, IHC, WB
Validations
  • (8)
Cat. No. ABIN1491388
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human, Mouse, Rat
Clonality Polyclonal
Application ELISA, IF (cc), IF (p), IHC (fro), IHC (p), WB
Validations
  • (3)
  • (2)
Cat. No. ABIN669951
Quantity 100 μL
Datasheet Datasheet
Reactivity Human, Mouse, Rat
Clonality Polyclonal
Application EIA, IHC (p), WB
Validations
  • (2)
Cat. No. ABIN500642
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human
Clonality Monoclonal
Application IA, IHC (fro), FACS, IHC (p), WB
Validations
  • (2)
Cat. No. ABIN2192047
Quantity 100 μg
Datasheet Datasheet
Reactivity Goat, Human, Mouse, Pig, Rat
Clonality Polyclonal
Application ELISA, FACS, IF (p), IHC (fro), IHC (p), WB
Validations
  • (14)
  • (6)
Cat. No. ABIN727205
Quantity 100 μL
Datasheet Datasheet
Reactivity Human, Mouse
Clonality Polyclonal
Application IHC (p), WB
Validations
  • (6)
  • (3)
Cat. No. ABIN2855448
Quantity 100 μL
Datasheet Datasheet

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Diabetic Macular Edema (DME)

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human, Rat
Clonality Monoclonal
Application ELISA, FACS, ICC, IHC, WB
Validations
  • (5)
Cat. No. ABIN5542567
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human, Mouse, Rat
Clonality Polyclonal
Application EIA, IHC (p), WB
Validations
  • (2)
Cat. No. ABIN500642
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Goat, Human, Mouse, Pig, Rat
Clonality Polyclonal
Application ELISA, FACS, IF (p), IHC (fro), IHC (p), WB
Validations
  • (14)
  • (6)
Cat. No. ABIN727205
Quantity 100 μL
Datasheet Datasheet

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Glaucoma

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human, Rat
Clonality Monoclonal
Application ELISA, FACS, ICC, IHC, WB
Validations
  • (5)
Cat. No. ABIN5542567
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human, Rat
Clonality Polyclonal
Application IHC (p), WB
Validations
  • (3)
Cat. No. ABIN2857031
Quantity 100 μL
Datasheet Datasheet
Reactivity Human, Mouse
Clonality Polyclonal
Application ELISA, ICC, IF, IHC, WB
Validations
  • (4)
Cat. No. ABIN6259810
Quantity 100 μL
Datasheet Datasheet

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Diabetic Retinopathy

Product
Reactivity
Clonality
Application
Validations
Cat. No.
Quantity
Datasheet
Reactivity Human
Clonality Polyclonal
Application IHC, IHC (p), WB
Validations
  • (1)
Cat. No. ABIN2616159
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human, Mouse, Rat
Clonality Polyclonal
Application ELISA, FACS, ICC, IF (cc), IF (p), IHC (fro), IHC (p), WB
Validations
  • (1)
  • (5)
Cat. No. ABIN730798
Quantity 100 μL
Datasheet Datasheet
Reactivity Human
Clonality Monoclonal
Application ICC, FACS, IHC, ELISA, WB
Validations
  • (2)
  • (8)
Cat. No. ABIN1098134
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human
Clonality Monoclonal
Application ELISA, FACS, IF, IHC, WB
Validations
  • (8)
Cat. No. ABIN1491388
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human, Mouse, Rat
Clonality Polyclonal
Application ELISA, IF (cc), IF (p), IHC (fro), IHC (p), WB
Validations
  • (3)
  • (2)
Cat. No. ABIN669951
Quantity 100 μL
Datasheet Datasheet
Reactivity Human, Mouse, Rat
Clonality Polyclonal
Application EIA, IHC (p), WB
Validations
  • (2)
Cat. No. ABIN500642
Quantity 0.1 mg
Datasheet Datasheet
Reactivity Human
Clonality Monoclonal
Application IA, IHC (fro), FACS, IHC (p), WB
Validations
  • (2)
Cat. No. ABIN2192047
Quantity 100 μg
Datasheet Datasheet
Reactivity Goat, Human, Mouse, Pig, Rat
Clonality Polyclonal
Application ELISA, FACS, IF (p), IHC (fro), IHC (p), WB
Validations
  • (14)
  • (6)
Cat. No. ABIN727205
Quantity 100 μL
Datasheet Datasheet
Reactivity Human, Mouse
Clonality Polyclonal
Application IHC (p), WB
Validations
  • (6)
  • (3)
Cat. No. ABIN2855448
Quantity 100 μL
Datasheet Datasheet

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References

Trapani, Auricchio: "Has retinal gene therapy come of age? From bench to bedside and back to bench." in: Human molecular genetics, Vol. 28, Issue R1, pp. R108-R118, (2020) (PubMed).

Gordon, Del Medico, Sander, Kumar, Hamad: "Gene therapies in ophthalmic disease." in: Nature reviews. Drug discovery, Vol. 18, Issue 6, pp. 415-416, (2019) (PubMed).

Lee, Wang, Chen, Li, Edwards, Hewitt, Liu: "Gene therapy for visual loss: Opportunities and concerns." in: Progress in retinal and eye research, Vol. 68, pp. 31-53, (2019) (PubMed).

Wang, Ling, Tu, van Wijngaarden, Dusting, Liu: "Gene therapy for diabetic retinopathy: Are we ready to make the leap from bench to bedside?" in: Pharmacology & therapeutics, Vol. 173, pp. 1-18, (2017) (PubMed).

Wang, Roberts, Liu: "Updates on Gene Therapy for Diabetic Retinopathy." in: Current diabetes reports, Vol. 20, Issue 7, pp. 22, (2020) (PubMed).

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