Abstract: The invention provides methods of treating Pompe's disease using human acid alpha glucosidase. A preferred treatment regime comprises administering greater than 10 mg/kg body weight per week to a patient.

Abstract: The invention provides transgenic nonhuman mammals expressing C1 inhibitor in their milk. The C1 inhibitor is useful in treating patients with hereditary angioedema or patients requiring immunosuppression.

Abstract: This invention relates to the production of recombinant proteins in mammals' milk. Particularly, this invention relates to an expression system which when transgenically incorporated into a mammal permits the female species of that mammal to produce the desired recombinant protein in or along with its milk. This invention also relates to the transgenic mammal that produces the desired recombinant product in its milk.

Abstract: The invention provides transgenic nonhuman mammals producing phosphorylated lysosomal proteins in their milk, and methods of generating the same. Phosphorylation occurs at the 6′ position of a mannose side chain residue. Also provided are methods of purifying lysosomal proteins from milk, and incorporating the proteins into pharmaceutical compositions for use in enzyme replacement therapy.

Abstract: The invention provides transgenic nonhuman mammals capable secreting exogenous procollagen or collagen into their milk. The mammals are healthy and capable of producing procollagen or collagen at high levels, usually in trimeric form. Suitable transgenes for incorporation into the mammals are also provided.

Abstract: The invention provides transgenic nonhuman mammals producing phosphorylated lysosomal proteins in their milk, and methods of generating the same. Phosphorylation occurs at the 6' position of a mannose side chain residue. Also provided are methods of purifying lysosomal proteins from milk, and incorporating the proteins into pharmaceutical compositions for use in enzyme replacement therapy.

Abstract: A process for the production of a peptide is disclosed, the process comprising expressing in the milk of a transgenic, non-human, placental mammal a fusion protein which comprises the peptide to be expressed linked to a fusion partner protein which is lysozyme. The fusion protein may be separate from the milk and cleaved to yield the target peptide. A transgenic, non-human, placental mammal whose genome incorporates a DNA molecule comprising a coding sequence encoding lysozyme coupled to a peptide is also described.

Abstract: Described are methods of targeting specific genes or DNA sequences encoding biologically active proteins to the mammary gland such that the DNA sequence is expressed in the mammary gland and the protein is secreted into the milk of the mammal. Also described is the production of transgenic nonhuman mammals whose germ line comprises a DNA gene complex containing a casein promoter, a casein enhancer, a DNA sequence which encodes a signal peptide and a DNA sequence encoding a biologically active protein. The milk containing the biologically active protein or the biologically active protein itself can be used in the making of diary products.

Abstract: Production of human procollagen or collagen in cells which ordinarily do not produce these molecules is effected by constructing expression systems compatible with mammary glands of non-human mammals. For example, expression systems can be microinjected into fertilized oocytes and reimplanted in foster mothers and carried to term in order to obtain transgenic non-human mammals capable of producing milk containing recombinant human procollagen or collagen. Human procollagen or collagen produced in this manner can be made of a single collagen type uncontaminated by other human or non-human collagens.

Abstract: Production of human procollagen or collagen in cells which ordinarily do not produce these molecules is effected by constructing expression systems compatible with mammary glands of non-human mammals. For example, expression systems can be microinjected into fertilized oocytes and reimplanted in foster mothers and carried to term in order to obtain transgenic non-human mammals capable of producing milk containing recombinant human procollagen or collagen. Human procollagen or collagen produced in this manner can be made of a single collagen type uncontaminated by other human or non-human collagens.

Abstract: The invention relates to the production of recombinant human fibrinogen (rhFib) in the milk of transgenic mammalian animals. This production and subsequent purification process is generally hampered by the occurrence of so-called ‘clots’ and ‘flakes’ in the milk which, in severe cases, may prevent the cow from being milked at all, resulting in a halt of lactation. These clots and flakes occur because of the expression of the fibrinogen protein, which is a factor that is normally involved in blood clotting. The invention relates to solving this milk clotting problem by treating the (lactating) animals with anticoagulants such as coumarins. A preferred anticoagulant that is used in the methods of the present invention is warfarin.

Abstract: The present invention relates to the use of a C1 inhibitor (C1INH) with shorter half-life than plasma-derived C1INH for the preparation of a medicament for the transient treatment of an individual. It relates to both therapeutic and prophylactic treatment. The method of the invention allows for the administration of C1INH at certain therapeutic levels for a concise pre-determined time span. Pharmaceutical compositions based on C1INH with shorter half-lives may be used both in situations where transient treatment is merely and advantage. The advantage of the use according to the invention is that an individual is not exposed to C1INH for longer than required, since the levels of the C1INH more rapidly subsides after administration has stopped. In contrast, levels of plasma-derived C1INH would remain elevated for a prolonged period of time.

Abstract: Transgenes for producing recombinant polypeptides transgenic bovine species. A transgene for producing recombinant polypeptides in the milk of transgenic bovine species comprises at least one expression regulation sequence, a secretory DNA sequence encoding a secretory signal sequence which is functional in mammary secretory cells of the bovine species and a recombinant DNA sequence encoding the recombinant polypeptide. Also included are methods for producing transgenic bovine species. The method includes introducing the above transgene into an embryonal target cell of a bovine species, transplanting the transgenic embryonic target cell formed thereby into a recipient bovine parent and identifying at least one female offspring which is capable of producing the recombinant polypeptide in its milk.

Abstract: Materials and methods for producing fibrinogen in transgenic non-human mammals are disclosed. DNA segments encoding A?, B? and ? chains of fibrinogen are introduced into the germ line of a non-human mammal, and the mammal or its female progeny produces milk containing fibrinogen expressed from the introduced DNA segments. Non-human mammalian embryos and transgenic non-human mammals carrying DNA segments encoding heterologous fibrinogen polypeptide chains are also disclosed.

Abstract: The present invention relates to the use of a C1 inhibitor (C1INH) with shorter half-life than plasma-derived C1INH for the preparation of a medicament for the transient treatment of an individual. It relates to both therapeutic and prophylactic treatment. The method of the invention allows for the administration of C1INH at certain therapeutic levels for a concise pre-determined time span. Pharmaceutical compositions based on C1INH with shorter half-lives may be used both in situations where transient treatment is merely and advantage. The advantage of the use according to the invention is that an individual is not exposed to C1INH for longer than required, since the levels of the C1INH more rapidly subsides after administration has stopped. In contrast, levels of plasma-derived C1INH would remain elevated for a prolonged period of time.