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Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells
Anai Gonzalez-Cordero1, Debbie Goh1, Kamil Kruczek1
11 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom .
Human Gene Therapy
|March 28, 2018
Summary
Adeno-associated virus (AAV) vectors show promise for retinal gene therapy. This study evaluated AAV serotype efficiency in human stem cell-derived retinal cells, identifying optimal vectors for preclinical research.
Area of Science:
- Ophthalmology
- Gene Therapy
- Stem Cell Biology
Background:
- Adeno-associated viral (AAV) vectors are promising for retinal gene therapy.
- Current efficacy evaluations rely heavily on animal models.
- Human pluripotent stem cell-derived retinal organoids offer an in vitro platform for testing.
Purpose of the Study:
- To compare the transduction efficiency of six AAV serotypes in mouse and human stem cell-derived retinal pigment epithelium (RPE) and photoreceptor cells.
- To assess the utility of human stem cell-derived photoreceptors for promoter specificity studies.
- To guide AAV vector selection and design for retinal gene therapy preclinical evaluations.
Main Methods:
- Testing of six AAV serotypes (AAV2/2, AAV2/9, AAV2/8, AAV2/8T(Y733F), AAV2/5, and ShH10).
- Transduction efficiency assessment in mouse and human pluripotent stem cell-derived RPE and photoreceptor cells in vitro.
- Evaluation of human embryonic stem cell-derived photoreceptors for promoter specificity.
Main Results:
- All tested AAV serotypes transduced RPE and photoreceptor cells in vitro.
- AAV ShH10 and AAV2/5 demonstrated the highest efficiency in transducing both mouse and human RPE.
- AAV2/8 and ShH10 showed robust transduction of human embryonic stem cell-derived cone photoreceptors.
- Human embryonic stem cell-derived photoreceptors successfully established promoter specificity in vitro.
Conclusions:
- AAV ShH10 and AAV2/5 are efficient vectors for human RPE transduction.
- AAV2/8 and ShH10 are effective for human cone photoreceptor transduction.
- Human stem cell-derived retinal organoids are valuable tools for preclinical gene therapy assessment and vector optimization.
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