摘要:
The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.
摘要:
The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.
摘要:
The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.
摘要:
The present invention provides a recombinant human &agr;-L-iduronidase and biologically active fragments and mutants thereof, large scale methods to produce and purify commercial grade recombinant human &agr;-L-iduronidase enzyme as well as methods to treat certain genetic disorders including &agr;-L-iduronidase deficiency and mucopolysaccharidosis I (MPS 1).
摘要:
The present invention relates to the use of receptor-associate protein (RAP) and fragments and variants thereof to improve delivery of therapeutic compounds to the liver and provides methods to treat liver disorders and conditions, such as hepatic carcinoma, by administering RAP or RAP variants conjugated to active agents.
摘要:
This invention provides compositions of highly phosphorylated lysosomal enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases.
摘要:
Polynucleotide sequences encoding enzymes that possess .alpha.2-3 neuraminidase activity are provided. Of particular interest are polynucleotide sequences encoding an enzyme having .alpha.2-3 neuraminidase activity and naturally produced by the bacteria Streptococcus pneumoniae. Recombinant DNA expression of enzymes possessing .alpha.2-3 specific neuraminidase activity is also described, including methods, recombinant host cells and a genetic construction. The invention also provides a purified enzyme having .alpha.2-3 neuraminidase activity from Streptococcus pneumoniae, wherein the enzyme is isolated from S. pneumoniea cultures. Another aspect of this invention is to provide methods of isolating genes encoding enzymes having neuraminidase activity, particularly .alpha.2-3 neuraminidase activity. The gene isolation methods of the invention comprise the step of labeling a hybridization probe derived from the neuraminidase coding portion of plasmid pND-1. The hybridization probe may then be used to isolate homologous genes encoding enzymes having the desired enzymatic activity.
摘要:
The present invention relates generally to receptor-selective variants of the low-density lipoprotein receptor-associated protein (RAP) and compositions thereof, methods of generating such variants and methods of using such receptor-selective RAP variant compositions for therapeutic purposes. The invention also relates to antibodies that bind to one or a small subset of CR-containing proteins.