Nonintegrating Gene Therapy Vectors
Gene delivery vectors that do not rely on host cell genome integration offer several advantages for gene transfer, chiefly the avoidance of insertional mutagenesis and position effect variegation. However, unless…
Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy
Gene therapy using hematopoietic stem cells (HSC) has developed over the past 3 decades, with progressive improvements in the efficacy and safety. Autologous transplantation of HSC modified with murine gammaretroviral…
Gene Therapy
Daniel E. Bauer, MD, PhD, Editor Related posts: In Vivo Hematopoietic Stem Cell Transduction Therapeutic Gene Editing Safety and Specificity Opening Marrow Niches in Patients Undergoing Autologous Hematopoietic Stem Cell Gene…
Childhood Immune Thrombocytopenic Purpura: Diagnosis and Management
Immune thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by a low circulating platelet count caused by destruction of antibody-sensitized platelets in the reticuloendothelial system. ITP can be classified as…
Hydroxyurea for Children with Sickle Cell Disease
Hydroxyurea therapy offers promise for ameliorating the clinical course of children with sickle cell disease (SCD). Hydroxyurea is a prototypic therapeutic option; it can be administered with minimal side effects,…
Advances in Hemophilia: Experimental Aspects and Therapy
This article describes recent clinical and research advances in hemophilia therapy. Different prophylactic regimens for the management of severe hemophilia are described along with the use of adjuvant treatment options…
Update on Thalassemia: Clinical Care and Complications
β-Thalassemia, originally named Cooley anemia, is an inherited blood disease. Various types of thalassemia are inherited anemias caused by mutations at the globin gene loci on chromosomes 16 and 11,…
Oral Iron Chelators
Effective chelation therapy can prevent or reverse organ toxicity related to iron overload, yet cardiac complications and premature death continue to occur, largely related to difficulties with compliance in patients…
Pediatric Arterial Ischemic Stroke
Arterial ischemic stroke (AIS) is a rare disorder in children. Research suggests that risk factors, outcomes, and presentation are different from those of adult stroke. In particular, prothrombotic abnormalities and…
Cancer Immunotherapy: Will Expanding Knowledge Lead to Success in Pediatric Oncology?
The past 25 years have seen an increase in our understanding of immunology and further expansion in the clinical use of immunotherapeutic modalities. How immunotherapy will be integrated with chemotherapy,…
