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A new study shows that we may be able to boost the grafting ability in cells for people receiving a bone marrow transplant.

Allogenic stem cell transplantation is a treatment approach in people with diseases that are destructive to the bone marrow, such as multiple myeloma, leukemia, and Hodgkin´s lymphoma. The goal is to restore bone marrow after total-body chemotherapy and irradiation. The transplantation procedure involves transferring hematopoietic stem cells from compatible, healthy donors to a patient. An important complication of this procedure is rejection and destruction of the donated stem cells by the recipient’s immune system before engraftment can occur.

New clinical applications in regenerative medicine are motivating an intense interest in easily accessible sources for producing human stem cells. The 2007 breakthrough showing that induced pluripotent stem cells (iPSC) could be generated directly from adult cells has been a boon to the industry. Since that time, there has been a steady refinement of the cell culture techniques that guide the cell reprogramming process.

Stem cells are the foundation from which all other cells with specialized functions are generated. Stem cells are primarily found in the bone marrow, where they are produced, but can be induced to migrate into the peripheral blood circulation through a process known as mobilization.

An independent publication cites the use of bone marrow and mobilized peripheral blood sourced from HemaCare in a study aimed at improving the efficiency of resource consumption in gene therapy.

The publication, published in the journal Molecular Therapy, focuses on the use of lentiviral vector (LV) to introduce corrected versions of defective genes into stem cells. The goal of gene therapy is to transplant these stem cells into people suffering from genetic disease, in hopes of restoring the function of the faulty gene. One of the main challenges in this clinical strategy is the cost; substantial amounts of LV are needed to modify a therapeutically effective dose of stem cells.

In an independent publication ^[1] researchers at UCLA cited using leukopaks obtained from HemaCare in order to investigate a new gene editing method.

The group is researching strategies to treat genetic blood disorders such as sickle cell anemia. While there has been a great deal of research focused on using CRISPR/Cas9 gene therapy to treat these illnesses ^[2], current strategies for gene editing in hematopoietic stem cells are inefficient, and thus costly.