This presentation highlights the therapeutic possibilities of Axiomer platform that are not limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to engineer proteins or modify their function as well as creating de novo mutations.
… Ho 1 , Artur V Cideciyan 2 , Samuel G Jacobson 2 , Stephen R Russell 3 , Arlene V Drack 3 , Bart P Leroy 4, 5, 6 , Michael R Schwartz 7 , Wilhelmina den Hollander 7 , Hsin Yi Huang van … Hospital of Philadelphia, Philadelphia, Pennsylvania; 7 ProQR Therapeutics, Leiden, the Netherlands. Ho A, Cideciyan …
… visual acuity, and full-field stimulus testing. Stephen R. Russell 1 , Arlene V. Drack 1 , Artur V. Cideciyan 2 , … Zaeytijd 3 , Aniz Girach 6 , Wil den Hollander 6 , Michael R. Schwartz 6 , David M. Rodman 6 1 Department of … Thomas Jefferson University, Philadelphia, PA, USA; 6 ProQR Therapeutics, Leiden, the Netherlands. Russell SR, …
… visual acuity, and full-field stimulus testing. Stephen R. Russell 1 , Arlene V. Drack 1 , Artur V. Cideciyan 2 , … Zaeytijd 3 , Aniz Girach 6 , Wil den Hollander 6 , Michael R. Schwartz 6 , David M. Rodman 6 1 Department of … Thomas Jefferson University, Philadelphia, PA, USA; 6 ProQR Therapeutics, Leiden, the Netherlands. Video-recorded …
