This presentation reviews Axiomer editing oligonucleotides (EONs) structure activity, the potential of Axiomer platform as well as its applicability for the treatment of liver originated disorders.
The presentation reviews EONs structure activity, the potential of the Axiomer platform as well as its applicability for the treatment of liver originated disorders. Presented at TIDES USA on May 9, 2023 by Gerard Platenburg - ProQR Therapeutics.
… type 10 (LCA10) ARVO 2021 presentation by Stephen R. Russell from Iowa University on the Safety and efficacy of … Leber Congenital Amaurosis type 10. Author(s) Stephen R. Russell 1 , Arlene V. Drack 1 , Artur V. Cideciyan 2 , … The Children’s Hospital of Philadelphia, PA, USA; 5 ProQR Therapeutics, Leiden, the Netherlands …
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 …
