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US5082670A
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Method of grafting genetically modified cells to treat defects, disease or damage or the central nervous system
Assignee: University of California
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Filing: 1988-12-15
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Granted: 1992-01-21
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Expires: 2008-12-15
Abstract
Abstract Methods of genetically modifying donor cells by gene transfer for grafting into the central nervous system to treat diseased or damaged cells are disclosed. The modified donor cells produce a molecule capable of affecting the recovery of cells in the CNS. Methods and vectors for carrying out gene transfer and grafting are described.
Inventors
- 👤 Fred H. Gage
- 👤 Michael B. Rosenberg
- 👤 Theodore Friedmann
Bibliographic Details
Application Number
—
Jurisdiction
US
Data Source
google_patents
Last Synchronized
2026-10-23 03:20
CPC Classifications
C; C12; C12N; C12N15/00; C12N15/09; C12N15/63; C12N15/79; C12N15/85; C12N15/86; A; A61; A61K; A61K38/00; A61K38/16; A61K38/17; A61K38/18; A61K38/1825; A61K48/00; C07; C07K; C07K14/00; C07K14/435; C07K14/475; C07K14/48; C12N9/00; C12N9/0004; C12N9/0071; C12N9/10; C12N9/1025; C12N9/1029; C12Y; C12Y114/00; C12Y114/16; C12Y114/16002; C12N2740/00; C12N2740/00011; C12N2740/10011; C12N2740/13011; C12N2740/13041; C12N2740/13043; Y; Y10; Y10S; Y10S435/00; Y10S435/8215; Y10S435/948
Claims Analysis
Total Claims: 29
Claim 1
Independent Claim
56 words
A method for treating defective, diseased or damaged cells in the mammalian central nervous system comprising grafting donor cell from the same mammalian species into the central nervous system, said donor cells genetically modified to produce a functional molecule in a sufficient amount to ameliorate said defective, diseased or damaged cells in the central nervous system.
Claim 2
Independent Claim
56 words
A method for treating defective, diseased or damaged cells in the mammalian central nervous system comprising grafting donor cell from the same mammalian species into the central nervous system, said donor cells genetically modified to produce a functional molecule in a sufficient amount to ameliorate said defective, diseased or damaged cells in the central nervous system.
Claim 3
Dependent Claim
24 words
The method of claim 1 wherein the step of grafting said donor cells comprises introducing said donor cells into the brain of a subject.
Claim 4
Dependent Claim
25 words
The method of claim 1 wherein the step of grafting said donor cells comprises introducing said donor cells into the spinal cord of a subject.
Claim 5
Dependent Claim
19 words
The method of claim 2 or 3 wherein said introducing comprises in tracerebral, intraventricular, subdural space or intravenous injection.
Claim 6
Dependent Claim
20 words
The method of claim 1 wherein said donor cells are modified by insertion of a therapeutic transgene into said cells.
Claim 7
Dependent Claim
24 words
The method of claim 5 wherein said step of insertion comprises inserting a vector carrying said transgene, wherein said vector is a viral vector.
Claim 8
Independent Claim
12 words
The method of cliam 1 wherein sid vector is herpes virus vector.
Claim 9
Independent Claim
12 words
The method of cliam 1 wherein sid vector is herpes virus vector.
Claim 10
Dependent Claim
12 words
The method of claim 6 wherein said vector is a retroviral vector.
Claim 11
Dependent Claim
23 words
The method of claim 8 wherein said retroviral vector is the retroviral vector pLN.8RNL having a final construction as shown in FIG. 12.
Claim 12
Dependent Claim
23 words
The method of claim 8 wherein said retroviral vector is the retroviral vector pLTHRNL having a final construction as shown in FIG. 16.
Claim 13
Dependent Claim
22 words
The method of claim 5 wherein said step of insertion into donor cells comprises nonviral physical transfection of DNA encoding a transgene.
Claim 14
Dependent Claim
17 words
The method of claim 11 wherein said nonviral physical transfection comprises microinjection of DNA encoding a transgene.
Claim 15
Dependent Claim
15 words
The method of claim 5 wherein said step of insertion into donor cells comprises electroporation.
Claim 16
Dependent Claim
17 words
The method of claim 5 wherein said step of insertion into donor cells comprises chemically mediated transfection.
Claim 17
Dependent Claim
14 words
The method of claim 14 wherein said chemically mediated transfections comprises calcium phosphate transfection.
Claim 18
Dependent Claim
17 words
The method of claim 5 wherein said step of insertion into donor cells comprises liposomal mediated transfection.
Claim 19
Dependent Claim
32 words
The method of claim 1 wherein said molecule is selected from the group consisting of growth factors, enzymes, gangliosides, antibiotics, neurotransmitters, neurohormones, toxins, neurite promoting molecules, anti-metabolites and precursors of said molecules.
Claim 20
Dependent Claim
12 words
The method of claim 17 wherein said molecule is nerve growth factor.
Claim 21
Dependent Claim
11 words
The method of claim 17 wherein said molecule is tyrosine hydroxylase.
Claim 22
Dependent Claim
10 words
The method of claim 17 wherein said molecule is L-DOPA.
Claim 23
Dependent Claim
23 words
The method of claim 1 further comprising co-administration of a therapeutic agent for treating said disease or damage to the central nervous system.
Claim 24
Dependent Claim
32 words
The method of claim 21 wherein said therapeutic agent is selected from the group consisting of growth factors, gangliosides, antibiotics, neurotransmitlers, neurohormones, toxins, antimetabolites, neurite promoting molecules and precursors of these agents.
Claim 25
Dependent Claim
12 words
The method of claim 21 wherein said therapeutic agent is cellular matter.
Claim 26
Dependent Claim
26 words
The method of claim 23 wherein said cellular matter is selected from the group consisting of adrenal chromaffin cells, fetal brain tissue cells and placental cells.
Claim 27
Dependent Claim
27 words
The method of claim 1 further comprising implanting material to the site of said damage or disease, material to facilitate reconnection or ameliorative interactions of injured neurons.
Claim 28
Dependent Claim
34 words
The method of claim 25 wherein said material is selected from the group consisting of homogenate of brain, homogenate of placenta, whole cells, synthetic material, neurite promoting extracellular matrix, and genetically modified donor cells.
Claim 29
Dependent Claim
27 words
The method of claim 1 wherein said donor cells are selected from the group consisting of fibroblasts, neurons, glial cells, keratinocytes, hepatocytes, ependymal cells and chromaffin cells.
References Cited (Backward Citations)
US4497796A
Cited
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