Retroviral Vector Packaging Cell Lines and Purification Methods for Gene Therapy

This invention relates to a novel gammaretroviral vector packaging cell line and a method of producing gammaretroviral vectors suitable for gene therapy. The described vectors may contain the gibbon ape leukemia virus (GALV) envelope with a CD11D8 epitope tag enabling their purification on a monoclonal antibody conjugated column. These vectors have several advantages over existing systems, including a broader host range, higher infectivity, and lower potential for replication.

Detecting Levels of Chymotrypsin and Amylase using Rabbit Polyclonal Antibodies Generated from Purified Human Enzymes

The invention relates to rabbit antisera raised against purified human chymotrypsin and amylase. Both chymotrypsin and amylase are produced by the pancreas and play important roles in digestion. Abnormal levels of chymotrypsin and amylase have been known to occur with multiple pancreas-related disorders, including pancreatitis. Measuring levels of these two enzymes using these polyclonal antibodies can help determine if a pancreas is functioning correctly.

NIMH DAO Toolbox: Data acquisition software that enables real-time sample analysis

This technology relates to a software package called NIMH DAO Toolbox that uses multithreading and a unique buffer structure to shorten gaps in sample readouts. Data acquisition devices running in continuous sampling mode collect data samples at a given sampling rate. The samples are typically stored in a memory buffer and read out at a regular interval. If the sampling rate is short enough, there can be a gap between the time the first sample is acquired and the time that sample is available to the user. This gap is typically on the order of tens of milliseconds.

Development of monoclonal antibodies that detect specific forms of neurophysin bound to either vasopressin or oxytocin

This invention includes the generation and use of monoclonal antibodies that specifically recognize either arginine vasopressin (AVP) or oxytocin (OT) when bound to neurophysins. The neurophysins (NPs) are a family of proteins that bind to hormones as they are released from the hypothalamus and make their way to the pituitary gland. Monoclonal antibodies were generated that specifically recognize vasopressin bound to a neurophysin (NP-AVP) or oxytocin bound to a neurophysin (NP-OT). Seven monoclonal antibodies were characterized.

Transgenic mice useful for study of gonadotropin-releasing hormone (GnRH) and a GnRH-secreting neuronal cell line (GN cell line)

This technology involves the generation and use of a mouse model for studying hypogonadism in humans and a cell line to study cellular and molecular properties of gonadotropin-releasing hormone (GnRH) cells. The mouse model expresses the simian virus 40 T antigen driven by the GnRH promoter, resulting in hypogonadism due to an arrest in neuronal migration during development and tumor formation along the migratory pathway. Olfactory bulb tumors in this model animal were dispersed, and GnRH-secreting neuronal cell line (GN/NLT cell line) was established.

Synthesis and use of deuterated L-DOPS to treat norepinephrine deficiency

This invention relates to the synthesis and methods of using a drug, deuterated L-DOPS, to treat deficiencies in the neurotransmitter norepinephrine. This classic neurotransmitter has roles in both the brain and the periphery. In the brain, norepinephrine is thought to play important roles in attention, memory, sleep, pain, movement, distress, and mood. Outside the brain, norepinephrine mediates regulation of the circulation by the sympathetic nervous system by increasing blood pressure.

Diagnosing and Treating Collagen type VI-related Dystrophies Based on a New COL6A1 Mutation

This invention includes the identification of a new mutation in the collagen type VI (COL6A1) gene, including a method for diagnosing and treating patients with this mutation. Collagen type VI-related dystrophies (COL6-RD) are devastating neuromuscular disorders that manifest with progressive generalized muscle weakness, contractures, and respiratory failure. Currently, no cure exists for COL6-RD.

Treatment of Immune-mediated Brain Swelling with Combined Anti-LFA1/VLA4 Therapy

This technology includes a therapeutic approach to prevent secondary edema after cerebrovascular hemorrhage. Using an animal model, we found that edema is triggered by massive extravasation of myelomonocytic cells from the blood into the brain in response to hemorrhaging vessels. Administration of anti-LFA1 and anti-VLA4 antibodies resulted in an inhibition of extravasation of the myelomonocytic cells. This single dose treatment prevented secondary edema and markedly improved functional outcomes if administered within the first six hours following cerebrovascular hemorrhage.

Longer-lived Mouse Models for Studying Gaucher Disease

The invention is a novel longer-lived mouse model for Gaucher disease. Gaucher disease is a genetic disorder that results from deficiencies in the enzyme glucocerebrosidase (GBA). The use of animal models to study how the disease progresses has been invaluable in research of this disorder. However, existing mouse models have been limited due to early mortality because the GBA enzyme plays an important role in lysosomal storage.