Ophthalmic Translational Research Group
Head: Bence György
Translating science and technology to the clinic
Recent advances in the understanding of genes, cell types and circuits involved in vision opened up the possibility to develop repair strategies, including gene therapy, for the loss of vision. Gene therapy for vision loss is now a clinical reality with the first approved product based on adeno-associated viral vectors.
Today, gene editing nucleases and base editors allow us to manipulate genes with efficiency and precision we have never dreamed of. Light sensitivity of blind retinas can be restored using optogenetic sensors, and human retinal organoids allow us to test gene therapy in vitro in disease models resembling the human retina.
Our group uses these novel technologies to modulate or take control over biological processes and bring them to patients in need as therapeutics. Clinical translation involves continuous interaction between basic researchers, technology specialists, clinicians, regulatory specialists, patient advocacy organizations and the patients themselves.
We act as a bridge between IOB’s Molecular and Clinical Center as well as our patients to allow rapid translation of scientific results into therapy.
Current projects:
Base Editing for Stargardt Disease
Group Members
Group Leader: Bence György
The team members are working on different translational projects:
View them on the respective translational project pages for
Base Editing for Stargardt Disease
Selected Journal Articles
High levels of AAV vector integration into CRISPR-induced DNA breaks
Hanlon KS, Kleinstiver BP, Garcia SP, Zaborowski MP, Volak A, Spirig SE, Muller A, Sousa AA, Tsai SQ, Bengtsson NE, Lööv C, Ingelsson M, Chamberlain JS, Corey DP, Aryee MJ, Joung JK, Breakefield XO, Maguire CA, György B (2019)
Nature Communications September 2019. PubMed PMID: 31570731 ;10(1):4439
Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss
György B, Nist-Lund C, Pan B, Asai Y, Karavitaki KD, Kleinstiver BP, Garcia SP, Zaborowski MP, Solanes P, Spataro S, Schneider BL, Joung JK, Géléoc GSG, Holt JR, Corey DP(2019)
Nat Med. 2019 Jul;25(7):1123-1130. PubMed PMID: 31270503
György B, Meijer EJ, Ivanchenko MV, Tenneson K, Emond F, Hanlon KS, Indzhykulian AA, Volak A, Karavitaki KD, Tamvakologos PI, Vezina M, Berezovskii VK, Born RT, O'Brien M, Lafond JF, Arsenijevic Y, Kenna MA, Maguire CA, Corey DP (2018)
Mol Ther Methods Clin Dev. 2018 Nov 20;13:1-13. doi: 10.1016/j.omtm.2018.11.003. eCollection 2019 Jun 14. PubMed PMID: 30581889; PubMed Central PMCID: PMC6297893
György B, Lööv C, Zaborowski MP, Takeda S, Kleinstiver BP, Commins C, Kastanenka K, Mu D, Volak A, Giedraitis V, Lannfelt L, Maguire CA, Joung JK, Hyman BT, Breakefield XO, Ingelsson M (2018)
Mol Ther Nucleic Acids. 2018 Jun 1;11:429-440. doi: 10.1016/j.omtn.2018.03.007. Epub 2018 Mar 16. PubMed PMID: 29858078; PubMed Central PMCID: PMC5992788
Wassmer SJ, Carvalho LS, György B, Vandenberghe LH, Maguire CA (2017)
Sci Rep. 2017 Mar 31;7:45329. doi: 10.1038/srep45329. PubMed PMID: 28361998; PubMed Central PMCID: PMC5374486.