Abstract: MO-1 is a newly identified gene and gene product associated with morbid obesity. Isolated MO-1 nucleic acids, MO-1 polypeptides, oligonucleotides that hybridize to MO-1 nucleic acids, and vectors, including expression vectors, comprising MO-1 nucleic acids are disclosed, as are isolated host cells, antibodies, transgenic non-human animals, compositions, and kits relating to MO-1. Methods of detecting the presence of MO-1 nucleic acid, methods of screening for agents which affect MO-1 activity, and methods of screening for MO-1 variants are also disclosed.

Abstract: MO-1 is a newly identified gene and gene product associated with morbid obesity. Isolated MO-1 nucleic acids, MO-1 polypeptides, oligonucleotides that hybridize to MO-1 nucleic adds, and vectors, including expression vectors, comprising MO-1 nucleic acids are disclosed, as are isolated host cells, antibodies, transgenic non-human animals, compositions, and kits relating to MO-1. Methods of detecting the presence of MO-1 nucleic acid, screening for agents which affect MO-1 activity, and screening for MO-1 variants are also disclosed.

Abstract: The present invention provides a method for treating individuals affected with the acid sphingomyelinase-deficient forms of Niemann-Pick disease (i.e., Type A or Type B Niemann-Pick) by administering small molecules as specific molecular “chaperones” for the deficient acid sphingomyelinase (ASM) enzyme associated with the disease. The molecules are ceramide, sphingomyelin, or phosphonucleotide analogues.