Loading patent…
AssigneeYeda Research and Development Co. Ltd.
InventorsAnthony Futerman, Joel Sussman, Israel Silman, Michal Harel, Hay Dvir, Lilly Toker, Swetlana Adamsky
Estimating the term…
A method of identifying a compound capable of correcting an impaired enzymatic activity of a mutant glucocerebrosidase molecule, the method comprising: (a) obtaining a first set of structure coordinates, the first set of structure coordinates defining a 3D structure of a glucocerebrosidase molecule capable of displaying normal enzymatic activity or a portion thereof; (b) computationally generating using the first set of structure coordinates a second set of structure coordinates, the second set of structure coordinates defining a predicted 3D structure of the mutant glucocerebrosidase molecule or a portion thereof; and (c) computationally identifying, using the second set of structure coordinates, a compound capable of interacting with the mutant glucocerebrosidase molecule in such a way as to correct the impaired enzymatic activity thereof, thereby identifying the compound capable of correcting the impaired enzymatic activity of the mutant glucocerebrosidase molecule. A glucocerebrosidase preparation comprising a population of glucocerebrosidase molecules, wherein substantially each of said glucocerebrosidase molecules: (i) has an amino acid sequence at least 95 percent homologous to an amino acid sequence set forth by SEQ ID NO: 1 or 8; (ii) is glycosylated at, or has an aspartatic acid residue at, glycosylation residue 1 of said amino acid sequence; and (iii) is independently unglycosylated at one or more glycosylation residues selected from the group consisting of glycosylation residues 2, 3 and 4 of said amino acid sequence.
1-122. (canceled)
A composition-of-matter comprising a crystallized glucocerebrosidase molecule, wherein said crystallized glucocerebrosidase molecule is characterized by an X-ray diffraction capacity enabling generation of a set of structure coordinates defining a 3D structure of said glucocerebrosidase molecule or a portion thereof.
A glucocerebrosidase preparation comprising a population of glucocerebrosidase molecules, wherein substantially each of said glucocerebrosidase molecules:
A method of producing a glucocerebrosidase preparation suitable for treatment of a disease associated with glucocerebrosidase deficiency, the method comprising exposing a plurality of glucocerebrosidase molecules to conditions suitable for partial deglycosylation thereof so as to form a population of partially deglycosylated glucocerebrosidase molecules each characterized by an amino acid sequence:
A method of identifying a compound capable of correcting an impaired enzymatic activity of a mutant glucocerebrosidase molecule, the method comprising:
A computing platform capable of generating a model representing a 3D structure of a glucocerebrosidase molecule or a portion thereof, the computing platform comprising:
A computer-readable medium comprising, in a retrievable format, data including a set of structure coordinates defining a 3D structure of a glucocerebrosidase molecule or a portion thereof, wherein said set of structure coordinates defines said 3D structure at a resolution of 2.9 angstroms or higher, and/or wherein an amino acid sequence of said glucocerebrosidase molecule is partially glycosylated.
A computer generated model representing a 3D structure of a glucocerebrosidase molecule or a portion thereof, wherein the model represents said glucocerebrosidase molecule or a portion thereof at a resolution of 2.9 angstroms or higher, and/or wherein an amino acid sequence of said glucocerebrosidase molecule is partially glycosylated.
Dependencies are inferred from the claim text.