EP0446017A1
Expired / Lapsed
EP
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New diagnostic and treatment methods involving the cystic fibrosis transmembrane regulator
Assignee: Genzyme Corp
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Filing: 1991-03-05
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Granted: —
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Expires: 2011-03-05
Abstract
Abstract Disclosed are full length isolated DNAs encoding cystic fibrosis transmembrane conductance regulator (CFTR) protein and a variety of mutants thereof. Also disclosed are antibodies specific for various CFTR domains and methods for their production. Expression of CFTR from cells transformed with these CFTR genes or cDNAs demonstrate surprising CFTR intracellular distributions and results thereby providing for new diagnostic and therapeutic procedures.
Inventors
- 👤 Richard J. Gregory
- 👤 Seng H. Cheng
- 👤 Alan Smith
- 👤 Sucharita Paul
- 👤 Kathleen M. Hehir
- 👤 John Marshall
Bibliographic Details
Application Number
—
Jurisdiction
EP
Data Source
google_patents
Last Synchronized
2026-11-08 01:57
CPC Classifications
C; C07; C07K; C07K16/00; C07K16/18; A; A61; A61K; A61K9/00; A61K9/10; A61K9/127; A61K9/1271; A61P; A61P11/00; B; B01; B01D; B01D61/00; C07K14/00; C07K14/435; C07K14/46; C07K14/47; C07K14/4701; C07K14/4712; A01; A01K; A01K2217/00; A01K2217/05; A61K38/00; A61K48/00
Claims Analysis
Total Claims: 30
Claim 1
Independent Claim
22 words
An isolated DNA comprising a nucleic acid sequence coding for a molecule having the biological activity of cystic fibrosis transmembrane conductance regulator.
Claim 2
Dependent Claim
15 words
The DNA of claim 1 comprising a cDNA coding for cystic fibrosis transmembrane conductance regulator.
Claim 3
Dependent Claim
21 words
The cDNA of claim 2 further comprising at least one intron located within the cystic fibrosis transmembrane conductance regulator coding region.
Claim 4
Dependent Claim
28 words
The cDNA of claim 2 further comprising phage, viral, liposome or virosome elements for enabling introduction of the cDNA encoding cystic fibrosis transmembrane conductance regulator into a cell.
Claim 5
Dependent Claim
16 words
The cDNA of claim 2 comprising the amino acid encoding sequence set forth in Table 1.
Claim 6
Dependent Claim
15 words
The cDNA of claim 5 further comprising at least one element for stabilizing the cDNA.
Claim 7
Dependent Claim
19 words
The DNA of claim 1 in a low copy number vector comprising DNA encoding cystic fibrosis transmembrane conductance regulator.
Claim 8
Dependent Claim
29 words
The DNA of claim 7 comprising a cDNA comprising at least one intron located within the amino acid encoding region of cystic fibrosis transmembrane conductance regulator in vector pkk-CFTR3.
Claim 9
Dependent Claim
30 words
A therapeutic composition the phage, virus, liposome or virosome comprising the isolated DNA sequence of claim 1 capable of effecting the production of cystic fibrosis transmembrane conductance regulator in humans.
Claim 10
Dependent Claim
25 words
A host cell comprising the DNA of claim 1, the cDNA of claim 2, the cDNA of claim 7, or the DNA of claim 8.
Claim 11
Dependent Claim
28 words
Cystic fibrosis transmembrane conductance regulator isolated from the mammalian host cell comprising purified DNA or single cDNA sequence encoding a protein having cystic fibrosis transmembrane conductance regulator activity.
Claim 12
Dependent Claim
18 words
The cystic fibrosis transmembrane conductance regulator of claim 11 wherein the host cell is that of claim 10.
Claim 13
Dependent Claim
23 words
A therapeutically effective composition for treating cystic fibrosis comprising the cystic fibrosis transmembrane conductance regulator isolated from the host cell of claim 10.
Claim 14
Dependent Claim
26 words
The therapeutically effective composition of claim 13 further comprising a carrier for delivering the composition to cells requiring augmentation of cystic fibrosis transmembrane conductance regulator function.
Claim 15
Dependent Claim
15 words
The therapeutically effective composition of claim 14 which is introduced intranasally or by repiratory aerosol.
Claim 16
Dependent Claim
49 words
A method for treating a disease condition having the characteristics of cystic fibrosis comprising the step of administering to cells having defective cystic fibrosis transmembrane conductance regulator function a therapeutically effective dose of the DNA of claim 1, the cDNA of claim 2 or the cDNA of claim 8.
Claim 17
Dependent Claim
47 words
A method for treating a disease condition having the characteristics of cystic fibrosis comprising the step of administering to cells having defective cystic fibrosis transmembrane conductance regulator function a therapeutically effective dose of the cystic fibrosis transmembrane conductance regulator isolated from the host cell of claim 10.
Claim 18
Dependent Claim
51 words
A method for screening compositions for selecting therefrom compounds capable of affecting cystic fibrosis transmembrane conductance regulator function comprising the steps of contacting said compositions to be screened with the host cell of claim 10 and detecting those compounds which affect the cystic fibrosis transmembrane conductance regulator phenotype of said cell.
Claim 19
Dependent Claim
42 words
A method for producing cystic fibrosis transmembrane conductance regulator comprising the steps of culturing the host cells of claim 10 under conditions permitting expression of the cystic fibrosis transmembrane conductance regulator and isolating from said cells, said cystic fibrosis transmembrane conductance regulator.
Claim 20
Dependent Claim
24 words
A kit comprising cystic fibrosis transmembrane conductance regulator agent means produced by the host cells of claim 10 packaged in a container, and instructions.
Claim 21
Dependent Claim
25 words
A transgenic animal comprising the DNA of claim 1, the cDNA of claim 2, the DNA of claim 8, or the DNA of claim 5.
Claim 22
Dependent Claim
13 words
An antibody specific for an epitope of the cystic fibrosis transmembrane conductance regulator.
Claim 23
Dependent Claim
96 words
A method for detecting the presence of the cystic fibrosis transmembrane conductance regulator in a biological sample comprising the steps of: a) contacting said biological sample of claim 22 which is a monoclonal antibody under conditions conducive to permit immunological complexes to form, b) allowing the monoclonal antibody to bind to the cystic fibrosis transmembrane conductance regulator to form an immunological complex, and c) detecting the formation of said immunological complex and correlating the presence or absence of said immunological complex with the presence or absence of cystic fibrosis transmembrane conductance regulator in the biological sample.
Claim 24
Dependent Claim
60 words
A method for obtaining purified cystic fibrosis transmembrane conductance regulator from an impure solution containing said regulator comprising the steps of: a) contacting said impure solution with the antibody of claim 22 which is a monoclonal antibody, b) allowing the monoclonal antibody to bind to said regulator to form a complex, and c) separating the complex from the impure solution.
Claim 25
Dependent Claim
36 words
The antibody of claim 22 wherein the epitope is selected from the group consisting of Exon 13, Exon 1, Exon 10, Exon 24 and extracellular loops approximately defined by amino acids 139-194 and amino acids 881-911.
Claim 26
Independent Claim
45 words
A method for producing antibodies specific for CFTR comprising the steps of forming a fusion protein comprising a first protein and a polypeptide comprising at least one CFTR domain, employing said fusion protein as an immunogen and collecting antibodies formed in response to said immunogen.
Claim 27
Dependent Claim
11 words
The method of claim 26 wherein said first protein is β-galactosidase.
Claim 28
Independent Claim
36 words
A method for diagnosing cystic fibrosis transmembrane conductance regulator dysfunction in mammalian host cell comprising the step of identifying the presence or absence of band C of cystic fibrosis transmembrane conductance regulator isolated from such cells.
Claim 29
Dependent Claim
35 words
The method of claim 28 which further comprises identifying the amount of non-glycosylated and partially glycosylated cystic cystic fibrosis transmembrane conductance regulator associated with said cell and correlating said amounts with cystic fibrosis genetic mutations.
Claim 30
Independent Claim
59 words
A method for reducing cystic fibrosis transmembrane conductance regulator dysfunction resulting from excessive presence or activity thereof in non-plasma membrane locations in cystic fibrosis cells comprising administrating an effective amount of an agent for deactivating the non-plasma membrane located cystic fibrosis transmembrane conductance regulator or causing the transport of said cystic fibrosis transmembrane conductance regulator to the plasma membrane.
References Cited (Backward Citations)
WO1991002796A1
Cited
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