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Infantile hemangiomas, otherwise known as infantile capillary hemangiomas, strawberry hemangiomas, or strawberry nevi, are nonmalignant vascular tumors that commonly affect children. The natural disease course typically involves growth for up to a year, followed by regression without treatment over a period of years with no cosmetic or functional sequelae. Less commonly, however, infantile hemangiomas can become a threat to vision or even life depending on location and size of the lesion. In addition, infantile hemangiomas, particularly those involving the face, may be disfiguring and result in lifelong sequelae. β-blockers have become a mainstay of therapy given their relatively low-risk profile and efficacy. Other treatment modalities previously described in the literature include corticosteroids (both intralesional and systemic), imiquimod, vincristine, bleomycin A5, and interferon α. More recently, angiotensin-converting enzyme inhibitors such as captopril have been used. Laser therapy and, less commonly, surgical excision are also available treatment options. We review current recommended management and treatment of capillary hemangiomas and discuss the benefits and risks of all previously reported treatment modalities. [ABSTRACT FROM AUTHOR]

Peripheral nerve sheath tumors of the orbit and ocular adnexa are a rare group of neoplasms hallmarked by nonspecific clinical presentations, variable tumor locations, challenging therapeutic efforts, and occasional diagnostic dilemmas. We review these tumor types and provide an updated summary on their clinical, histopathologic, radiological, and emerging molecular features. [ABSTRACT FROM AUTHOR]

Sickle cell disease (SCD) can be cured by allogeneic hematopoietic stem cell transplant. However, this is only possible when a matched donor is available, making the development of gene therapy using autologous hematopoietic stem cells a highly desirable alternative. We used a culture model of human erythropoiesis to directly compare two insulated, self-inactivating, and erythroid-specific lentiviral vectors, encoding for γ-globin (V5m3-400) or a modified β-globin (βAS3-FB) for production of antisickling hemoglobin (Hb) and correction of red cell deformability after deoxygenation. Bone marrow CD34 + cells from three SCD patients were transduced using V5m3-400 or βAS3-FB and compared with mock-transduced SCD or healthy donor CD34 + cells. Lentiviral transduction did not impair cell growth or differentiation, as gauged by proliferation and acquisition of erythroid markers. Vector copy number averaged approximately one copy per cell, and corrective globin mRNA levels were increased more than sevenfold over mock-transduced controls. Erythroblasts derived from healthy donor and mock-transduced SCD cells produced a low level of fetal Hb that was increased to 23.6 ± 4.1% per vector copy for cells transduced with V5m3-400. Equivalent levels of modified normal adult Hb of 17.6 ± 3.8% per vector copy were detected for SCD cells transduced with βAS3-FB. These levels of antisickling Hb production were sufficient to reduce sickling of terminal-stage red blood cells upon deoxygenation. We concluded that the achieved levels of fetal Hb and modified normal adult Hb would likely prove therapeutic to SCD patients who lack matched donors. [ABSTRACT FROM AUTHOR]