Expression system for effeiciently producing clinically effective lysosomal enzymes (glucocerebrosidase)
OwnerSusan L Berentinventor
Life of this patent
Estimating the term…
Abstract
The invention as described herein relates to the efficient production of recombinant, clinically effective lysosomal enzymes using a transformed insect cell expression system. For example, to create the expression system of the invention, any insect cell can be transfected with a plasmid comprised of a gene encoding the human glucocerebrosidase gene and genetic elements that enhance its expression. The insect cell transfected with the plasmid encoding glucocerebrosidase secretes synthesized glucocere-brosidase into its growth media. The recombinantly produced clinically effective glucocerebrosidase produced by the insect cell expression system can be used to treat Gaucher's disease.
Claims52
A pharmaceutical composition comprising clinically effective recombinant glucocerebrosidase synthesized with an expression system comprised of an insect cell transformed with a vector encoding glucocerebrosidase, wherein the expression system synthesizes clinically effective glucocerebrosidase.
A method for treating individuals with deficiencies in glucocerebrosidase, wherein the method includes introducing into individuals with deficiencies in glucocerebrosidase recombinantly produced glucocerebrosidase from insect cells, wherein the recombinantly produced glucocerebrosidase is produced by insect cells in a clinically effective form.
An expression system comprised of an insect cell transformed with a vector encoding glucocerebrosidase that synthesizes clinically effective glucocerebrosidase.
A method of producing clinically effective recombinant glucocerebrosidase comprising the steps of:
- (a) developing a vector that encodes a gene for glucocerebrosidase;
- (b) introducing the vector into at least one insect cell that is capable of receiving the vector and serving as host to the vector;
- (c) nurturing the at least one insect cell comprising the vector so that the glucocerebrosidase gene of the vector is successfully transcribed and translated into glucocerebrosidase in its clinically effective form;
- and
- (d) harvesting the glucocerebrosidase.
A method of producing clinically effective recombinant glucocerebrosidase comprising the steps of:
- (a) creating a vector encoding a gene for glucocerebrosidase with a signal sequence for secretion functionally linked to an enhancer and a promoter, wherein the vector also encodes a structural gene that increases transcription as well as a structural gene that is a detectable marker;
- (b) introducing the vector into an insect cell;
- (c) growing the transformed insect cell under conditions so that it can optimally synthesize and secrete clinically effective glucocerebrosidase and create a multitude of cells that can synthesize and secrete clinically effective glucocerebrosidase;
- and
- (d) collecting recombinantly synthesized clinically effective glucocerebrosidase from the extracellular environment.
Dependencies are inferred from the claim text.