摘要:
The present invention provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.
摘要:
The present invention provides devices, small interfering RNAs, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient.
摘要:
Methods and kits are provided for determining which single nucleotide polymorphism (“SNP”) variant of an allele of a heterozygous patient is on the same mRNA transcript as a disease-causing mutation that is at a remote region of the gene's mRNA comprising a) an allele-specific reverse transcription reaction using an allele-specific primer, and b) analysis of the resulting cDNA product from the reverse transcription reaction at the region of the mutation to determine the presence or absence of the mutation on this allele-specific cDNA product.
摘要:
An intrathecal catheter for delivering fluid to, or withdrawing fluid from, the cerebrospinal fluid compartment of a patient, includes a proximal end and a distal portion. The distal portion has an outer diameter of about 1 mm or less, a bending stiffness of about 0.002 pounds per square inch or less, and a distal end. A delivery region is located between the proximal end and the distal end, and a lumen extends from the proximal end to the delivery region. The catheter may further include a coiled structure region to prevent dislodgement of the catheter from a tissue in which the coiled structure is implanted, for example the cisterna magna. The catheter may also include tines to anchor portions of the catheter, for example to a portion of the spinal canal.
摘要:
A microsyringe includes a body, a barrier movable within the body, and a flow regulator. The body includes a pressurizable chamber, an inlet fluidly connected to the pressurizable chamber, an outlet fluidly connected to the pressurizable chamber, and a cannula fluidly connected to the outlet. The flow regulator is fluidly connected to the cannula to limit the flow of a pharmaceutical through the cannula to a maximum rate. The movable barrier fluidly separates the inlet from the outlet. A pharmaceutical is disposed in a space between the movable barrier and the outlet. The inlet is designed to mechanically and fluidly couple to a hydraulic fluid delivery system. Hydraulic fluid from the inlet pressurizes the chamber, causing the barrier to move and displace the pharmaceutical out of the chamber and into the cannula. The flow regulator ensures that the flow rate of the pharmaceutical through the cannula is not excessive. The movable barrier may be a piston or a collapsible membrane.
摘要:
An implantable antiarrhythmia device which detects and classifies arrhythmias of the human heart, and delivers appropriate therapy. The device employs a method of arrhythmia classification based on a set of prioritized rules, each of the rules defining a plurality of criteria based upon characteristics of sensed depolarizations of heart tissue, each rule being met when the criteria associated with the rule are met. Some rules, when met, trigger delivery of antiarrhythmia therapy. Other rules, when met, inhibit delivery of antiarrhythmia therapy. The rules may be met simultaneously, and if so, the highest priority rule governs the behavior of the device.
摘要:
Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatto and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease.
摘要:
The invention provides novel combination therapies for treating Huntington's disease which comprise a) BDNF or suitable fragments thereof and b) agents capable of causing inhibition of a gene responsible for the neurodegenerative disease. The invention provides nucleic acid sequences, methods, and systems suitable for applications of these combination therapies.
摘要:
The present invention provides devices, small interfering RNAs, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient.
摘要:
The invention provides novel combination therapies for treating Huntington's disease which comprise a) BDNF or suitable fragments thereof and b) agents capable of causing inhibition of a gene responsible for the neurodegenerative disease. The invention provides nucleic acid sequences, methods, and systems suitable for applications of these combination therapies.