Safety and Tolerability Study of an Intravenously Administered Small Interfering Ribonucleic Acid (siRNA) Post On-Pump Cardiothoracic Surgery in Patients at Risk of Acute Kidney Injury

Autor: Segav Demirjian, Gorav Ailawadi, Martin Polinsky, Dani Bitran, Shuli Silberman, Stanton Keith Shernan, Michel Burnier, Marta Hamilton, Elizabeth Squiers, Shai Erlich, Daniel Rothenstein, Samina Khan, Lakhmir S. Chawla

Publikováno v: Kidney International Reports, Vol 2, Iss 5, Pp 836-843 (2017)

Patients undergoing on-pump cardiac surgery are at an increased risk of acute kidney injury. QPI-1002, a small interfering ribonucleic acid, is under clinical development for the prevention of acute kidney injury. The safety, tolerability, and pharma

Externí odkaz: https://doaj.org/article/f74d25425ae648c4aa53f8b008ae7f02

Safety and Tolerability Study of an Intravenously Administered Small Interfering Ribonucleic Acid (siRNA) Post On-Pump Cardiothoracic Surgery in Patients at Risk of Acute Kidney Injury

Autor: Lakhmir S. Chawla, Segav Demirjian, Shai Erlich, Gorav Ailawadi, Samina Khan, Martin Polinsky, Marta Hamilton, Shuli Silberman, Stanton K. Shernan, Elizabeth C. Squiers, Dani Bitran, Michel Burnier, Daniel Rothenstein

Publikováno v: Kidney international reports, vol. 2, no. 5, pp. 836-843
Kidney International Reports
Kidney International Reports, Vol 2, Iss 5, Pp 836-843 (2017)

Introduction Patients undergoing on-pump cardiac surgery are at an increased risk of acute kidney injury. QPI-1002, a small interfering ribonucleic acid, is under clinical development for the prevention of acute kidney injury. The safety, tolerabilit

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::53b07989eac680fccff3044ff333d6d6
https://doi.org/10.1016/j.ekir.2017.03.016

Fluorescence-Based Selection of Gene-Corrected Hematopoietic Stem and Progenitor Cells From Acid Sphingomyelinase-Deficient Mice: Implications for Niemann-Pick Disease Gene Therapy and the Development of Improved Stem Cell Gene Transfer Procedures

Autor: Arie Dagan, Shai Erlich, Jan W.M. Visser, Edward H. Schuchman, Silvia R.P. Miranda, Shimon Gatt

Publikováno v: Blood. 93:80-86

The general utility of a novel, fluorescence-based procedure for assessing gene transfer and expression has been demonstrated using hematopoietic stem and progenitor cells. Lineage-depleted hematopoietic cells were isolated from the bone marrow or fe

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5df640606e80ddcdb3ef5b9e8ce12344
https://doi.org/10.1182/blood.v93.1.80

Bone Marrow Transplantation in Acid Sphingomyelinase-Deficient Mice: Engraftment and Cell Migration Into the Brain as a Function of Radiation, Age, and Phenotype

Autor: Arie Dagan, V L Friedrich, Jan W.M. Visser, Shai Erlich, Silvia R.P. Miranda, Shimon Gatt, Edward H. Schuchman

Publikováno v: Blood. 90:444-452

Types A and B Niemann-Pick disease (NPD) result from the deficient activity of the lysosomal hydrolase, acid sphingomyelinase (ASM). A long-term goal of our research is to evaluate the effects of bone marrow transplantation (BMT) and hematopoietic st

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4de9a1e3b735bfc31c7f87ca4df7d52f
https://doi.org/10.1182/blood.v90.1.444

Toxicological and pharmacokinetic properties of chemically modified siRNAs targeting p53 RNA following intravenous administration

Autor: Douglas J. Kornbrust, Shai Erlich, Elisabeth C.R. Solano, James D. Thompson, Bruce A. Molitoris, David J. Schneider, Jeffrey W.-D. Foy, Elena Feinstein

Publikováno v: Nucleic acid therapeutics. 22(4)

We report the toxicological and pharmacokinetic properties of the synthetic, small interfering RNA I5NP following intravenous administration in rodents and nonhuman primates. I5NP is designed to act via the RNA interference (RNAi) pathway to temporar

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::61a47bece8fbb8a62c4992fcd8d2701b
https://pubmed.ncbi.nlm.nih.gov/22913596