An in vitro, light responsive, retinal model for accurate predictions of in vivo outcomes you can have confidence in.
Gene therapy in the retina
Most inherited retinal diseases (IRDs) cause blindness due to progressive deterioration of light-sensing photoreceptors in the retina and is a focus for retinal gene therapy. Gene therapy is becoming an emerging tool for the diseases unreachable by other therapies. The two key modes of application are via a subretinal or intravitreal injection of the therapeutic vector into the eye. Adeno Associated Virus (AAV) vectors have emerged as favorable tools for gene transfer into the retina. However, they still need to be improved to increase the efficiency of transduction of photoreceptors. Exploiting the ability of AAV serotypes displaying different abilities to deliver the desired genes into photoreceptors in combination with engineering through peptide insertions or amino acid substitutions in the capsid, new AAV vectors can be engineered, and robust in vitro model is needed to allow rapid evaluation of such new therapeutic vectors.
- Model for gene therapy using AAV: human photoreceptor-like cells within Newcells’ iPSC-derived retinal organoids are efficiently transduced with AAV, demonstrating suitability of the retina platform for preclinical testing and assessment of retinal gene therapy treatments (Submitted).
More information on the retina platform
A model is built using data of viral macromolecular structure from Protein Data Bank (PDB 3NG9)
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