This review focuses on the most recent data on biotherapeutic approaches, using DNA, RNA, recombinant proteins, or cells as therapeutic tools or targets for the treatment of neuromuscular diseases. Many of these novel technologies have now reached the clinical stage and have or are about to move to the market. Others, like genome editing are still in an early stage but hold great promise.
View Article and Find Full Text PDFInt J Environ Res Public Health
September 2011
Manufactured nanomaterials (MNs) are commonly considered to be commercial products possessing at least one dimension in the size range of 10(-9) m to 10(-7) m. As particles in this size range represent the smaller fraction of colloidal particles characterized by dimensions of 10(-9) m to 10(-6) m, they differ from both molecular species and bulk particulate matter in the sense that they are unlikely to exhibit significant settling under normal gravitational conditions and they are also likely to exhibit significantly diminished diffusivities (when compared to truly dissolved species) in environmental media. As air/water, air/soil, and water/soil intermedium transport is governed by diffusive processes in the absence of significant gravitational and inertial impaction processes in environmental systems, models of MN environmental intermedium transport behavior will likely require an emphasis on kinetic approaches.
View Article and Find Full Text PDFBiochem Biophys Res Commun
March 2005
Retroviral vectors are widely used tools for gene therapy. However, in vivo gene transfer is only effective in dividing cells, which, in liver, requires a regenerative stimulus. Follistatin is effective in promoting liver regeneration after 90% and 70% hepatectomy in rats.
View Article and Find Full Text PDFObjective: Because intrauterine transplantation of fetal hepatocytes could become an effective approach for treating severe genetic disorders of the liver, the objective of this study was to demonstrate the feasibility of in utero allotransplantation of fetal hepatocytes in a nonhuman primate model using direct intraparenchymal administration of donor cells.
Methods: Fetal primary hepatocytes were isolated from 3 fetal primates (MACACA MULATTA) at 89-120 days of gestation, and cryopreserved. When a recipient was available, the cells were thawed and transduced by a beta-galactosidase-expressing retrovirus (3 cases) or labelled with a fluorescent dye (4 cases).
Engineered retroviruses are widely used vectors for cancer gene therapy approaches. However, the ability to target cells of therapeutic interest while controlling the expression of the transferred genes would improve both the efficiency and the safety of viral vectors. In this study, we investigated the ability of a retroviral amphotropic envelope displaying single-chain variable-fragment (scFv) directed against the c-Met receptor, to target the entry of recombinant retroviruses to human hepatocarcinoma cells.
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