Abstract: The present invention generally provides improved gene therapy vectors, cell-based compositions, and methods of using the same in methods of gene therapy. The present invention further provides improved gene therapy compositions for expanding hematopoietic cells and related methods for treatment of diseases, disorders, and conditions of the hematopoietic system such as thalassemias and anemias.

Abstract: The present invention generally provides improved gene therapy vectors, cell-based compositions, and methods of using the same in methods of gene therapy. The present invention further provides improved gene therapy compositions for expanding hematopoietic cells and related methods for treatment of diseases, disorders, and conditions of the hematopoietic system such as thalassemias and anemias.

Abstract: The present invention generally provides improved gene therapy vectors, cell-based compositions, and methods of using the same in methods of gene therapy. The present invention further provides improved gene therapy compositions for expanding hematopoietic cells and related methods for treatment of diseases, disorders, and conditions of the hematopoietic system such as thalassemias and anemias.

Abstract: The present invention generally provides improved gene therapy vectors, cell-based compositions, and methods of using the same in methods of gene therapy. The present invention further provides improved gene therapy compositions for expanding hematopoietic cells and related methods for treatment of diseases, disorders, and conditions of the hematopoietic system such as thalassemias and anemias.

Abstract: The present invention provides cell compositions for in vivo implantation, and designed for the sustained and controlled delivery of therapeutic substances.

Abstract: The present invention provides a chemical class of active agents to be used as efficacious drugs in preventing erythrocyte dehydration, thus helping ameliorate the symptoms of certain hemoglobinopathies. The active agents include a class of compounds that can deliver to an erythrocyte in vivo a divalent cation. Increased intracellular concentrations of the divalent cation in the erythrocyte block K—Cl cotransport preventing dehydration of the erythrocyte, thus improving erythrocyte survival. These agents are to be administered by any preferred route of administration including oral, intravenous and parenteral routes. These agents may be co-administered with any other anti-hemoglobinopathic compounds. The methodology is effective for both long term and short term therapy; may be employed prophylactically and/or therapeutically; and may be used in acute crisis clinical situations.

Abstract: The device chiefly includes a thermostatically controlled measuring cell (1) with which is associated a transmitting-receiving ultrasonic probe (2). The cell consists of a cavity (10) divided half way up by a thin plate (11). The upper chamber above the plate is designed to receive a suspension (13) of particles while the probe is located in the lower chamber below the plate. The upper face (14) of the probe is separated from the plate.

Abstract: The invention relates to novel thiosulfonate derivatives of general formu a:R--S--SO.sub.2 --R.sub.1in which R represents a 1H-imidazol-2-yl, quinolin-2-yl, 5-amino-1,3,4-thiadizol-2-yl or 4,5-dihydro-thiazol-2-yl radical and R.sub.1 represents a lower alkyl, (hydroxy)lower alkyl, phenyl, (lower alkyl) phenyl, (hydroxy lower alkyl) phenyl, (lower alkanoyl) phenyl or quinolin-8-yl radical.The thiosulfonate derivatives in question exert an inhibitory action with respect to the malformation or destruction of red blood corpuscules due to a genetic modification of haemoglobin or to parasites and can, therefore, be used in the treatment of drepanocytosis, malaria and babebiosis.

Abstract: The invention relates to pharmaceutical or veterinary compositions for initing the malformation or destruction of red blood corpuscules due to a genetic modification of haemoglobin or to parasites, compositions comprising, as active ingredient, at least one thiosulfonate derivative of general formula:R--S--SO.sub.2 --R.sub.1in which R represents a 1-methyl-imidazol-2-yl, 1H-benzimidazol-2-yl or benzoxazol-2-yl radical and R.sub.1 represents a lower alkyl, (hydroxy) lower alkyl, lower alkanoyl, phenyl, (lower alkyl) phenyl, (hydroxy lower alkyl) phenyl, (lower alkanoyl) phenyl or quinolin-8-yl radical.The compositions in question can be used in the treatment of drepanocytosis, malaria and babebiosis.