AP2003002800A0 - Hematopoietic stem cell gene therapy - Google Patents
Hematopoietic stem cell gene therapyInfo
- Publication number
- AP2003002800A0 AP2003002800A0 APAP/P/2003/002800A AP2003002800A AP2003002800A0 AP 2003002800 A0 AP2003002800 A0 AP 2003002800A0 AP 2003002800 A AP2003002800 A AP 2003002800A AP 2003002800 A0 AP2003002800 A0 AP 2003002800A0
- Authority
- AP
- ARIPO
- Prior art keywords
- cells
- thymus
- patient
- hematopoietic stem
- lhrh
- Prior art date
Links
- 210000003958 hematopoietic stem cell Anatomy 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 210000004027 cell Anatomy 0.000 abstract 7
- 210000001541 thymus gland Anatomy 0.000 abstract 4
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 abstract 1
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 abstract 1
- 101000904173 Homo sapiens Progonadoliberin-1 Proteins 0.000 abstract 1
- 229940124041 Luteinizing hormone releasing hormone (LHRH) antagonist Drugs 0.000 abstract 1
- 102100024028 Progonadoliberin-1 Human genes 0.000 abstract 1
- 101000996723 Sus scrofa Gonadotropin-releasing hormone receptor Proteins 0.000 abstract 1
- 239000000556 agonist Substances 0.000 abstract 1
- 230000000735 allogeneic effect Effects 0.000 abstract 1
- 230000004069 differentiation Effects 0.000 abstract 1
- 239000012634 fragment Substances 0.000 abstract 1
- 239000003163 gonadal steroid hormone Substances 0.000 abstract 1
- XLXSAKCOAKORKW-UHFFFAOYSA-N gonadorelin Chemical compound C1CCC(C(=O)NCC(N)=O)N1C(=O)C(CCCN=C(N)N)NC(=O)C(CC(C)C)NC(=O)CNC(=O)C(NC(=O)C(CO)NC(=O)C(CC=1C2=CC=CC=C2NC=1)NC(=O)C(CC=1NC=NC=1)NC(=O)C1NC(=O)CC1)CC1=CC=C(O)C=C1 XLXSAKCOAKORKW-UHFFFAOYSA-N 0.000 abstract 1
- 210000003738 lymphoid progenitor cell Anatomy 0.000 abstract 1
- 230000001404 mediated effect Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 210000003643 myeloid progenitor cell Anatomy 0.000 abstract 1
- 230000007420 reactivation Effects 0.000 abstract 1
- 108020003175 receptors Proteins 0.000 abstract 1
- 230000010076 replication Effects 0.000 abstract 1
- 230000011664 signaling Effects 0.000 abstract 1
- 208000010648 susceptibility to HIV infection Diseases 0.000 abstract 1
- 229960005486 vaccine Drugs 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/36—Skin; Hair; Nails; Sebaceous glands; Cerumen; Epidermis; Epithelial cells; Keratinocytes; Langerhans cells; Ectodermal cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/08—Peptides having 5 to 11 amino acids
- A61K38/09—Luteinising hormone-releasing hormone [LHRH], i.e. Gonadotropin-releasing hormone [GnRH]; Related peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/39—Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/461—Cellular immunotherapy characterised by the cell type used
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/464838—Viral antigens
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/18—Antivirals for RNA viruses for HIV
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/04—Immunostimulants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
- A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the dose, timing or administration schedule
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2510/00—Genetically modified cells
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Immunology (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Mycology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Developmental Biology & Embryology (AREA)
- Genetics & Genomics (AREA)
- Hematology (AREA)
- Molecular Biology (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Wood Science & Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Endocrinology (AREA)
- Reproductive Health (AREA)
- Tropical Medicine & Parasitology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- AIDS & HIV (AREA)
- Transplantation (AREA)
Abstract
The present disclosure provides methods for gene therapy utilising hematopoietic stem cells, lymphoid progenitor cells, and/or myeloid progenitor cells. The cells are genetically modified to provide a gene that is expressed as in these cells and their progeny after differentiation. In a preferred embodiment the cells contain a gene or gene fragment that confers to the cells resistance to HIV infection and/or replication. The cells are administered to a patient in conjunction with treatment to reactivate the patient's thymus. The cells may be autologous, syngeneic, allogeneic or xenogeneic, as tolerance to foreign cells is created in the patient during reactivation of the thymus. In a preferred embodiment the hematopoietic stem cells are CD34+. The patient's thymus is reactivated by disruption of sex steroid mediated signalling to the thymus. In a preferred embodiment this disruption is created by administration of LHRH agonists, LHRH antagonists, anti-LHRH receptor antibodies, anti-LHRH vaccines or combinations thereof.
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US79530200A | 2000-10-13 | 2000-10-13 | |
| US79528600A | 2000-10-13 | 2000-10-13 | |
| AUPR0745A AUPR074500A0 (en) | 2000-10-13 | 2000-10-13 | Treatment of t cell disorders |
| US75891001A | 2001-01-10 | 2001-01-10 | |
| US09/966,576 US20020071833A1 (en) | 1999-04-15 | 2001-09-26 | Hematopoietic stem cell gene therapy |
| US96951001A | 2001-10-01 | 2001-10-01 | |
| PCT/IB2001/002739 WO2002031110A2 (en) | 2000-10-13 | 2001-10-12 | Hematopoietic stem cell gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AP2003002800A0 true AP2003002800A0 (en) | 2003-06-30 |
Family
ID=27542982
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| APAP/P/2003/002800A AP2003002800A0 (en) | 2000-10-13 | 2001-10-12 | Hematopoietic stem cell gene therapy |
Country Status (10)
| Country | Link |
|---|---|
| EP (1) | EP1357978A4 (en) |
| JP (1) | JP2004511495A (en) |
| KR (1) | KR20030082542A (en) |
| CN (1) | CN1582178A (en) |
| AP (1) | AP2003002800A0 (en) |
| AU (1) | AU1632002A (en) |
| BR (1) | BR0114639A (en) |
| CA (1) | CA2462073A1 (en) |
| IL (1) | IL155413A0 (en) |
| WO (1) | WO2002031110A2 (en) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040258672A1 (en) * | 1999-04-15 | 2004-12-23 | Monash University | Graft acceptance through manipulation of thymic regeneration |
| JP2007518699A (en) * | 2003-04-18 | 2007-07-12 | ノーウッド・イミュノロジー・リミテッド | Disease prevention and vaccination before thymus regeneration |
| PL2250279T3 (en) | 2008-02-08 | 2016-11-30 | Anti-ifnar1 antibodies with reduced fc ligand affinity | |
| AU2011325833C1 (en) | 2010-11-05 | 2017-07-13 | Zymeworks Bc Inc. | Stable heterodimeric antibody design with mutations in the Fc domain |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU4236296A (en) * | 1994-11-10 | 1996-06-06 | Fred Hutchinson Cancer Research Center | Intrathymic stem cell implantation |
| AUPP977899A0 (en) * | 1999-04-15 | 1999-05-13 | Monash University | Improvement of t cell mediated immunity |
-
2001
- 2001-10-12 AU AU1632002A patent/AU1632002A/en active Pending
- 2001-10-12 AP APAP/P/2003/002800A patent/AP2003002800A0/en unknown
- 2001-10-12 JP JP2002534480A patent/JP2004511495A/en active Pending
- 2001-10-12 BR BR0114639-4A patent/BR0114639A/en not_active Application Discontinuation
- 2001-10-12 CN CNA018201385A patent/CN1582178A/en active Pending
- 2001-10-12 KR KR10-2003-7005255A patent/KR20030082542A/en not_active Application Discontinuation
- 2001-10-12 CA CA002462073A patent/CA2462073A1/en not_active Abandoned
- 2001-10-12 EP EP01986714A patent/EP1357978A4/en not_active Withdrawn
- 2001-10-12 IL IL15541301A patent/IL155413A0/en unknown
- 2001-10-12 WO PCT/IB2001/002739 patent/WO2002031110A2/en active Application Filing
Also Published As
| Publication number | Publication date |
|---|---|
| JP2004511495A (en) | 2004-04-15 |
| AU1632002A (en) | 2002-04-22 |
| KR20030082542A (en) | 2003-10-22 |
| WO2002031110A3 (en) | 2002-06-20 |
| IL155413A0 (en) | 2003-11-23 |
| CA2462073A1 (en) | 2002-04-18 |
| BR0114639A (en) | 2004-01-20 |
| EP1357978A2 (en) | 2003-11-05 |
| WO2002031110A2 (en) | 2002-04-18 |
| CN1582178A (en) | 2005-02-16 |
| EP1357978A4 (en) | 2004-05-19 |
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