Academic
Publications
Potential Large Animal Models for Gene Therapy of Human Genetic Diseases of Immune and Blood Cell Systems

Potential Large Animal Models for Gene Therapy of Human Genetic Diseases of Immune and Blood Cell Systems,Thomas R. Bauer,Rima L. Adler,Dennis D. Hick

Potential Large Animal Models for Gene Therapy of Human Genetic Diseases of Immune and Blood Cell Systems   (Citations: 5)
BibTex | RIS | RefWorks Download
Genetic mutations involving the cellular components of the hematopoietic system—red blood cells, white blood cells, and platelets—manifest clinically as anemia, infection, and bleed- ing. Although gene targeting has recapitulated many of these diseases in mice, these murine homologues are limited as translational models by their small size and brief life span as well as the fact that mutations induced by gene targeting do not always faithfully refl ect the clinical manifestations of such mutations in humans. Many of these limitations can be over- come by identifying large animals with genetic diseases of the hematopoietic system corresponding to their human disease counterparts. In this article, we describe human diseases of the cellular components of the hematopoietic system that have counterparts in large animal species, in most cases carrying mutations in the same gene (CD18 in leukocyte adhesion de- fi ciency) or genes in interacting proteins (DNA cross-link re- pair 1C protein and protein kinase, DNA-activated catalytic polypeptide in radiation-sensitive severe combined immuno- defi ciency). Furthermore, we describe the potential of these animal models to serve as disease-specifi c preclinical models for testing the effi cacy and safety of clinical interventions such as hematopoietic stem cell transplantation or gene therapy be- fore their use in humans with the corresponding disease.
Cumulative Annual
View Publication
The following links allow you to view full publications. These links are maintained by other sources not affiliated with Microsoft Academic Search.
Sort by: