Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy

Neural Plasticity
Carla Sanjurjo-Soriano, Vasiliki Kalatzis

Abstract

Inherited retinal dystrophies (IRDs) are a leading cause of visual impairment in the developing world. These conditions present an irreversible dysfunction or loss of neural retinal cells, which significantly impacts quality of life. Due to the anatomical accessibility and immunoprivileged status of the eye, ophthalmological research has been at the forefront of innovative and advanced gene- and cell-based therapies, both of which represent great potential as therapeutic treatments for IRD patients. However, due to a genetic and clinical heterogeneity, certain IRDs are not candidates for these approaches. New advances in the field of genome editing using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein (Cas) have provided an accurate and efficient way to edit the human genome and represent an appealing alternative for treating IRDs. We provide a brief update on current gene augmentation therapies for retinal dystrophies. Furthermore, we discuss recent advances in the field of genome editing and stem cell technologies, which together enable precise and personalized therapies for patients. Lastly, we highlight current technological limitations and barriers that need to be overcome b...Continue Reading

References

Feb 6, 1996·Proceedings of the National Academy of Sciences of the United States of America·Y G KimS Chandrasegaran
Sep 1, 1996·Molecular Medicine Today·L S Sullivan, S P Daiger
Feb 16, 2000·Progress in Retinal and Eye Research·J CaiD P Jones
Aug 15, 2000·Annual Review of Biophysics and Biomolecular Structure·S A WolfeC O Pabo
Apr 28, 2001·Nature Genetics·G M AclandJ Bennett
May 2, 2001·Nature Reviews. Neuroscience·M A Dyer, C L Cepko
Apr 16, 2002·Molecular Microbiology·Ruud JansenLeo M Schouls
Dec 12, 2003·Nature Reviews. Immunology·J Wayne Streilein
Aug 19, 2004·Cell·Katarzyna KozarPiotr Sicinski
Apr 14, 2006·Human Genetics·Kenneth H Warrington, Roland W Herzog
Aug 16, 2006·Proceedings of the National Academy of Sciences of the United States of America·Deepak A LambaThomas A Reh
Aug 16, 2006·American Journal of Human Genetics·Anneke I den HollanderFrans P M Cremers
Sep 22, 2006·Human Molecular Genetics·Hannie KremerRonald Roepman
Nov 23, 2006·Lancet·Dyonne T HartongThaddeus P Dryja
Feb 3, 2007·Orphanet Journal of Rare Diseases·Christian P Hamel
Mar 24, 2007·Science·Rodolphe BarrangouPhilippe Horvath
Jun 15, 2007·American Journal of Human Genetics·Mary O'ReillyG Jane Farrar
Jul 6, 2007·Experimental Neurology·Larry Benowitz, Yuqin Yin
Apr 29, 2008·The New England Journal of Medicine·Albert M MaguireJean Bennett
Apr 29, 2008·The New England Journal of Medicine·James W B BainbridgeRobin R Ali
Sep 24, 2008·Proceedings of the National Academy of Sciences of the United States of America·Artur V CideciyanWilliam W Hauswirth
Oct 16, 2008·Clinical Microbiology Reviews·Shyam Daya, Kenneth I Berns
Nov 5, 2008·The Journal of Cell Biology·Ravid ShechterMichal Schwartz
Jan 21, 2009·Cellular and Molecular Life Sciences : CMLS·B PardoA Aguilera
Jan 29, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Naomi ChaddertonG Jane Farrar
Apr 22, 2009·Neuroscience Letters·Yasuhiko HiramiMasayo Takahashi
Aug 27, 2009·Proceedings of the National Academy of Sciences of the United States of America·Jason S MeyerDavid M Gamm
Nov 12, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Zhijian WuPeter Colosi
Apr 7, 2010·Progress in Retinal and Eye Research·Wolfgang BergerJohn Neidhardt

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Citations

May 28, 2019·International Journal of Molecular Sciences·Michalitsa DiakatouVasiliki Kalatzis
Mar 1, 2020·International Journal of Molecular Sciences·Yuliya Markitantova, Vladimir Simirskii
Mar 17, 2020·Current Opinion in Ophthalmology·Dominic A PradoRamiro S Maldonado
Feb 3, 2021·European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society·A SuppiejA Leon
Feb 27, 2021·Seminars in Ophthalmology·Elizabeth J RossinLeo A Kim
Aug 11, 2020·Molecular Therapy. Methods & Clinical Development·Martijn P T ErnstW W M Pim Pijnappel
Mar 17, 2021·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Daria MamaevaVasiliki Kalatzis
Apr 4, 2021·International Journal of Molecular Sciences·Michalitsa DiakatouVasiliki Kalatzis
Oct 15, 2020·Journal of Ocular Pharmacology and Therapeutics : the Official Journal of the Association for Ocular Pharmacology and Therapeutics·Ratnesh K SinghIgor O Nasonkin
Jan 8, 2020·Molecular Therapy. Methods & Clinical Development·Carla Sanjurjo-SorianoVasiliki Kalatzis
Oct 19, 2021·Frontiers in Medicine·Jennifer Hernández-JuárezShyamanga Borooah

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Methods Mentioned

BETA
transgenic
gene knock-in
exome sequencing

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