Journal Article

TARGT Gene Therapy Platform for Correction of Anemia in End-Stage Renal Disease

Miari, Reem, Koren, Belly, Neil, Garry A, Lerner, Benny, Blum, Shany, Guzy, Serge, Efrati, Shai, Chernin, Gil, Nyska, Abraham, Shapir, Nir, Schwartz, Doron, Pergola, Pablo E, Doenyas-Barak, Keren, Yagil, Yoram

2017

Overview

A new protein-delivery platform can provide sustained secretion of recombinant human erythropoietin with the use of small, transduced autologous dermal implants. Study patients with end-stage renal disease were found to have maintenance of stable hemoglobin levels. To the Editor: Anemia in patients with end-stage renal disease who are undergoing hemodialysis is caused, in part, by a reduction in or absence of secretion of endogenous erythropoietin. Recombinant human erythropoietin given intravenously has been associated with an increased risk of cardiovascular complications and death, which is considered to be related, at least in part, to the high peak levels of plasma erythropoietin that are achieved after intravenous administration. 1 , 2 Transduced autologous restorative gene therapy (TARGT), a new autologous protein-delivery platform designed to provide sustained secretion of proteins such as recombinant human erythropoietin, uses a small autologous dermal explant . . .

• Publisher: Massachusetts Medical Society
• Subject: Anemia / Anemia - etiology / Anemia - therapy / Animals / Drug Implants / End-stage renal disease / Erythropoietin / Erythropoietin - blood / Erythropoietin - secretion / Gene therapy / Genetic Therapy - methods / Hemodialysis / Hemoglobin / Hemoglobins - analysis / Humans / Kidney diseases / Kidney Failure, Chronic - complications / Kidney Failure, Chronic - therapy / Mice / Patients / Renal Dialysis / Secretion / Skin