Abstract:
Broad cerebrospinal fluid (CSF) distribution of an agent is achievable by delivering the agent in a liquid formulation to the CSF at flow rates less than 500 microliters per hour, such as between about 2 microliters per hour and about 100 microliters per hour.
Abstract:
Methods, agents and devices for treating a patient to reduce accumulation of certain proteins in the brain are described. Such proteins include Aβ, and the methods, agents and devices are useful for reducing the accumulation of Aβ, which is a principal constituent of the plaques associated with such diseases as Alzheimer's disease (AD). Antibodies to Aβ, when delivered systemically or directly into the central nervous system, improve cognitive deficits in a transgenic mouse model of AD. However, unlike peripheral of antibodies to Aβ, which increased cerebral vascular plaques and hemorrhages, direct central administration did not result in such an increase cerebral vascular plaques and hemorrhages.
Abstract:
The instant invention provides a method for improving efficiency of RNA delivery to cells. The method comprises applying a low strength electric field to the cells and then after a certain time period, administering the ribonucleic acid sequence to the cells. Devices, kits, and RNA molecules suitable for delivery and devices suitable for practicing the disclosed methods are also provided.
Abstract:
The present invention is directed to compositions, methods and kits for regulation of gene therapies for Huntington's Disease, including, without limitation, reversible gene therapies and allele-specific therapies.
Abstract:
Liposome complexes are provided for site-specific delivery of pharmaceutical agents with improved targeting efficiency. The liposome complexes include a pharmaceutical agent associated with the liposome; a sialic acid-containing molecule associated with the liposome; and optionally a targeting agent attached to the sialic acid-containing molecule. The invention also provides methods of making liposome complexes and their in vivo administration.
Abstract:
The instant invention provides a method for improving efficiency of RNA delivery to cells. The method comprises applying a low strength electric field to the cells and then after a certain time period, administering the ribonucleic acid sequence to the cells. Devices, kits, and RNA molecules suitable for delivery and devices suitable for practicing the disclosed methods are also provided.
Abstract:
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.
Abstract:
Broad cerebrospinal fluid (CSF) distribution of an agent is achievable by delivering the agent in a liquid formulation to the CSF at flow rates less than 500 microliters per hour, such as between about 2 microliters per hour and about 100 microliters per hour.
Abstract:
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.
Abstract:
Methods, agents and devices for treating a patient to reduce accumulation of certain proteins in the brain are described. Such proteins include Aβ, and the methods, agents and devices are useful for reducing the accumulation of Aβ, which is a principal constituent of the plaques associated with such diseases as Alzheimer's disease (AD). Antibodies to Aβ, when delivered systemically or directly into the central nervous system, improve cognitive deficits in a transgenic mouse model of AD. However, unlike peripheral of antibodies to Aβ, which increased cerebral vascular plaques and hemorrhages, direct central administration did not result in such an increase cerebral vascular plaques and hemorrhages.