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7shRNA modified autologous stem cells

Development stage
Unknown
Lead developer
Chance Pharmaceuticals
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous, Intrathecal
01

Overview

7shRNA modified autologous stem cells are a form of cell therapy in which a patient's own stem cells are harvested, genetically modified ex vivo using seven short hairpin RNAs (shRNAs), and then reinfused into the patient. The shRNAs are designed to silence specific gene targets, potentially to correct disease-causing mutations or modulate cellular function for therapeutic benefit. This approach leverages the advantages of autologous transplantation—such as reduced risk of immune rejection and graft-versus-host disease—while adding targeted genetic modification through RNA interference. The primary mechanism involves both cell replacement/regeneration and gene silencing via RNA interference. Such therapies may be developed for various indications depending on the genes targeted by the shRNAs, including hematologic malignancies, neurodegenerative diseases, or other conditions where gene silencing in patient-derived stem cells could be beneficial[1][9].

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