Article

Non-Myelotoxic Agents as a Preparatory Regimen for Hematopoietic Stem Cell Gene Therapy

2023-07-18

Abstract excerpt

<title>Abstract</title> <p>RAG2 deficiency is characterized by a lack of B and T lymphocytes, causing severe lethal infections. Currently, RAG2 deficiency is treated with a Hematopoietic Stem Cell transplantation (HSCT). Most conditioning regimens used before HSCT consist of alkylating myelotoxic agents with or without irradiation and affect growth and development of pediatric patients. Here, we developed a non-m...

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Literature Corpus work: ffa18ecd-a778-5e24-ac8a-2c689b926c3f
DOI: 10.21203/rs.3.rs-3067174/v1

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