Title:
NON-INVASIVE DELIVERY OF POLYPEPTIDES THROUGH THE BLOOD-BRAIN BARRIER, AND IN VIVO SELECTION OF ENDOCYTOTIC LIGANDS
Document Type and Number:
WIPO Patent Application WO2003091387
Kind Code:
A9
Abstract:
A treatment method and genetic vectors are disclosed for non-invasive delivery of polypeptides through the blood brain barrier (BBB), to treat brain or spinal tissue. A genetic vector is used to transfect one or more neurons which "straddle" the BBB, such as sensory neurons, nocioceptive neurons, or lower motor neurons; this is done by administering the vector in a manner that causes it to contact neuronal projections that extend outside the BBB. Once inside a peripheral projection that belongs to a BBB-straddling neuron, the vectors (or some portion thereof) will be transported to the main cell body of the neuron, through a process called retrograde transport. Inside the main cell body, at least one gene carried by the genetic vector will be expressed, to form polypeptides. Some of these polypeptides (which can include leader sequences that will promote anterograde transport and secretion by BBB-straddling neurons) will be transported by the neurons to secretion sites inside the BBB. The polypeptides will be secreted by transfected neurons at locations inside the BBB, and will then contact and exert their effects upon secondary "target" neurons located entirely within the BBB. By using this system, polypeptides that stimulate nerve growth or activity can be used to treat neurodegenerative diseases, impaired limbs in stroke victims, etc., and polypeptides that suppress neuronal activity can be used to treat unwanted excessive neuronal activity, such as neuropathic pain. This approach also provides new methods for delivering endocrine and paracrine polypeptides into the CNS, thereby allowing improved medical and reproductive treatments in humans, and improved ability to modulate growth, maturation, reproduction, or other endocrine-related functions among livestock, endangered species, and other animals.
Inventors:
FERGUSON IAN A (AU)
TANI HIROAKI
TANI HIROAKI
Application Number:
PCT/IB2003/002371
Publication Date:
February 26, 2004
Filing Date:
April 28, 2003
Export Citation:
Assignee:
FERGUSON IAN A (AU)
International Classes:
C12N15/09; A61K9/127; A61K35/76; A61K38/00; A61K38/22; A61K39/395; A61K45/00; A61K47/46; A61K47/48; A61K48/00; A61P11/00; A61P25/00; C07K16/00; C07K16/28; C12N15/12; C12N15/16; C12N15/18; C12N15/861; C12N15/867; C12N15/869; (IPC1-7): A61K48/00; A61P11/00; C12N15/12; C12N15/16; C12N15/18; C12N15/79; G01N33/74
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