Abstract: A composition of matter comprising non-cellular particles, which comprise a lysosomal enzyme and/or a small molecule which increases an amount and/or activity of a lysosomal enzyme.

Abstract: Methods and kits for treating Gaucher disease are provided. The methods are based on using agents capable of inhibiting proteasomal degradation of glucocerebrosidase and/or elevating a level of mis-folded yet active glucocerebrosidase in cell lysosomes. Also provided are methods and kits for diagnosing and/or assessing a severity and determining prognosis of Gaucher disease or other diseases associated with abnormally folded proteins which are retained in the ER.

Abstract: The present invention teaches novel Eps15 homology (EH) domain containing proteins, polynucleotide sequences encoding the novel EH domain containing proteins, oligonucleotides and oligonucleotide analogs derived from the polynucleotide sequences, a display library displaying short peptides derived from the EH domain containing proteins, antibodies recognizing the EH domain containing proteins, peptides or peptide analogs derived from the EH domain containing proteins, and pharmaceutical compositions and methods of employing the peptides or peptide analogs, the oligonucleotides and oligonucleotide analogs, and/or the polynucleotide sequences to up-regulate or down-regulate clathrin coated pit mediated endocytosis and thereby insulin growth factor 1 receptor (IGF1 receptor) signaling.

Abstract: The present invention teaches novel Eps15 homology (EH) domain containing proteins, polynucleotide sequences encoding the novel EH domain containing proteins, oligonucleotides and oligonucleotide analogs derived from the polynucleotide sequences, a display library displaying short peptides derived from the EH domain containing proteins, antibodies recognizing the EH domain containing proteins, peptides or peptide analogs derived from the EH domain containing proteins, and pharmaceutical compositions and methods of employing the peptides or peptide analogs, the oligonucleotides and oligonucleotide analogs, and/or the polynucleotide sequences to up-regulate or down-regulate clathrin coated pit mediated endocytosis and thereby insulin growth factor 1 receptor (IGF1 receptor) signaling.