Work

Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony

Year: 2023

Type: article

Abstract: Recent advances in genome engineering have unlocked the potential of hematopoietic stem cell (HSC) gene therapy for various genetic disorders including β-hemoglobinopathies, immunodeficiency disorders, and blood-related cancers.1 Ensuring efficient engraftment of engineered cells is crucial in these approaches, making it essential to maximize cell infusion numbers while preserving their stemness and functionality. To achieve successful transplantation, robust stem cell collection methods and pre-transplant conditioning regimens are of utmost importance.

Source: Molecular Therapy — Methods & Clinical Development

DOI: 10.1016/j.omtm.2023.08.005

Authors: Selami Demirci, John F. Tisdale

Institutions: National Institutes of Health, National Heart, Lung, and Blood Institute

Language: English

Topic: Hematopoietic Stem Cell Transplantation

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900