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Vagni P, Perlini LE, Chenais NAL, et al. Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration. Front Neurosci. 2019;13:945doi: 10.3389/fnins.2019.00945.
Vagni, P., Perlini, L. E., Chenais, N. A. L., Marchetti, T., Parrini, M., Contestabile, A., Cancedda, L., & Ghezzi, D. (2019). Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration. Frontiers in neuroscience, 13945. https://doi.org/10.3389/fnins.2019.00945
Vagni, Paola, et al. "Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration." Frontiers in neuroscience vol. 13 (2019): 945. doi: https://doi.org/10.3389/fnins.2019.00945
Vagni P, Perlini LE, Chenais NAL, Marchetti T, Parrini M, Contestabile A, Cancedda L, Ghezzi D. Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration. Front Neurosci. 2019 Sep 10;13:945. doi: 10.3389/fnins.2019.00945. eCollection 2019. PMID: 31551698; PMCID: PMC6748340.
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Front Neurosci. 2019 Sep 10;13:945. doi: 10.3389/fnins.2019.00945. eCollection 2019.

Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration.

Frontiers in neuroscience

Paola Vagni, Laura E Perlini, Naïg A L Chenais, Tommaso Marchetti, Martina Parrini, Andrea Contestabile, Laura Cancedda, Diego Ghezzi

Affiliations

  1. Medtronic Chair in Neuroengineering, Center for Neuroprosthetics and Institute of Bioengineering, School of Engineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland.
  2. Laboratory of Local Micro-environment and Brain Development, Department of Neuroscience and Brain Technologies, Istituto Italiano di Tecnologia, Genoa, Italy.
  3. Dulbecco Telethon Institute, Roma, Italy.

PMID: 31551698 PMCID: PMC6748340 DOI: 10.3389/fnins.2019.00945

Abstract

Inherited retinal dystrophies (IRDs) are a large and heterogeneous group of degenerative diseases caused by mutations in various genes. Given the favorable anatomical and immunological characteristics of the eye, gene therapy holds great potential for their treatment. Our goal is to validate the preservation of visual functions by viral-free homology directed repair (HDR) in an autosomal recessive loss of function mutation. We used a tailored gene editing system based on clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) to prevent retinal photoreceptor death in the retinal degeneration 10 (Rd10) mouse model of retinitis pigmentosa. We tested the gene editing tool

Keywords: gene editing; in vivo electroporation; photoreceptors; retinal degeneration; vision

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