Drug intelligence / Profile preview

LN-neo transduced autologous CD34+ cells

Development stage
Phase 2
Lead developer
National Institutes of Health
Modality
Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

LN-neo transduced autologous CD34+ cells represent a historical milestone in ex vivo gene therapy, specifically utilized for gene marking studies during the early 1990s. This product involves the collection of a patient's own CD34+ hematopoietic stem and progenitor cells, which are then genetically modified using a retroviral vector (the LN or LNL6 vector, derived from the Moloney murine leukemia virus) to carry the bacterial neomycin phosphotransferase (*neo*) gene. Once the modified cells are re-infused into the patient—typically following high-dose chemotherapy or as part of a bone marrow transplant—the *neo* gene serves as a unique genetic barcode. This allows researchers to track the long-term survival, proliferation, and differentiation of the transplanted cells into various blood lineages by detecting the transgene or its protein product. These studies were primarily designed to investigate hematopoietic recovery and the source of relapse in cancer patients, as well as to validate the safety and efficiency of retroviral gene transfer technology in a clinical setting.

Other names
Neomycin-phosphotransferase-transduced autologous CD34+ cellsAutologous CD34+ cells transduced with LNL6 vectorNeo-marked autologous hematopoietic stem cells
02

Targets

16S rRNA (16S ribosomal RNA)

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