Cell and gene therapies using haematopoietic stem cells (HSCs) epitomize the
transformative potential of regenerative medicine. Recent clinical successes for gene …

The HIV latent reservoir represents the major challenge to cure development. Residing in
resting CD4+ T cells and myeloid cells at multiple locations in the body, including sanctuary …

Background Allogeneic hematopoietic stem-cell transplantation for X-linked severe
combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T …

X-linked severe immunodeficiency disease (SCID-X1) is an inherited, rare, and life-threating
disease. The genetic origin is a defect in the interleukin 2 receptor γ chain (IL2RG) gene and …

Retroviruses are obligate intracellular parasites that must integrate a copy of the viral
genome into the host DNA. The integration reaction is performed by the viral enzyme …

Fanconi anemia (FA) is a rare inherited disease that is associated with bone marrow failure
and a predisposition to cancer. Previous clinical trials emphasized the difficulties that …

In past two decades the gene therapy using genetic modified autologous hematopoietic
stem cells (HSCs) transduced with the viral vector has become a promising alternative …

As HSC GT is becoming available for more diseases, novel developments should focus on
improving availability while reducing costs of the treatment. Continued follow up of treated …

Abstract Purpose of Review Mobilized peripheral blood is the predominant source of stem
and progenitor cells for hematologic transplantation. Successful transplant requires sufficient …

The phenotypic spectrum of X-linked severe combined immunodeficiency (X-SCID) ranges
from typical X-SCID (early-onset disease in males that is fatal if not treated with …