11 Výsledek
Rhizomelic chondrodysplasia punctata (RCDP) is a group of rare diseases that have no known cure. It is a genetic disorder characterized by mutations in the Peroxisomal Biogenesis Factor 7 (PEX7) gene (RCDP1), Glyceronephosphate O-acyltransferase (GNPAT) gene (RCDP 2), Alkylglycerone phosphate
Alzheimer's disease (AD) is currently the 7th leading cause of death in the United States with approximately 5.9 million Americans living with the progressive and debilitating condition. AD predominantly affects older individuals as 86% of AD cases are in individuals 65 years of age and older.
Background and Rationale:
Locally relapsed prostate cancer presents an attractive target for gene therapy because the tumour remains localised to the prostate or to the surrounding tissue for a long phase of the disease. Also the delivery of gene therapy vectors to the prostate is straightforward
The Philadelphia chromosome-negative chronic myeloproliferative neoplasms (MPNs) are a group of hematopoietic stem cell malignancies that include polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF). PV and ET can evolve into myelofibrosis, termed post PV/ET MF.
The two most important causes of iron overload disease in humans are the iron-loading disorder hereditary hemochromatosis (HC), and transfusional siderosis in patients with chronic hematological diseases like myelodysplastic syndrome (MDS), thalassemia, and leukemia.
Hemochromatosis. In HC the
Bacterial and fungal infections in neutropenic patients or in patients with inherited disorders of neutrophil function continue to cause substantial morbidity and mortality. In particular, fungal infections are an increasingly important cause of death in patients receiving aggressive chemotherapy,
Hypoparathyroidism (HypoPT) is a relatively rare disease, where the level of parathyroid hormone (parat hormone [PTH]) cannot maintain a physiologically sufficient concentration of plasma calcium (P-Ca). This may be due to neck surgery (most frequently), congenital HypoPT, auto-immune destruction of
The success of BMT as a curative option for patients with malignancies is frequently limited by the inability to identify an appropriate donor in time for transplantation.
Transplantations utilizing umbilical cord blood stem cells are increasingly successful. Data suggest that there are fewer and
Background
Sarcoidosis is a multi-system granulomatous inflammatory disease. Pulmonary involvement is most common. Patients typically experience fatigue, weakness and dyspnea. Respiratory muscle weakness, which may be secondary to granulomatous inflammation, is associated with dyspnea and decreased
Selection of the Regimen for Immunosuppressive Therapy
Cyclophosphamide with ATG is a common conditioning regimen with two decades of experience in the treatment of aplastic anemia, and has been used safely without reported mortality in the treatment of autoimmune diseases such as systemic lupus