HRP20110386T1 - Sintetička sekvenca mecp2 za zamjensku proteinsku terapiju - Google Patents
Sintetička sekvenca mecp2 za zamjensku proteinsku terapiju Download PDFInfo
- Publication number
- HRP20110386T1 HRP20110386T1 HR20110386T HRP20110386T HRP20110386T1 HR P20110386 T1 HRP20110386 T1 HR P20110386T1 HR 20110386 T HR20110386 T HR 20110386T HR P20110386 T HRP20110386 T HR P20110386T HR P20110386 T1 HRP20110386 T1 HR P20110386T1
- Authority
- HR
- Croatia
- Prior art keywords
- protein
- mecp2
- fragment
- derivative
- polypeptide according
- Prior art date
Links
- 102000004169 proteins and genes Human genes 0.000 title claims abstract 18
- 108090000623 proteins and genes Proteins 0.000 title claims abstract 17
- 238000002560 therapeutic procedure Methods 0.000 title abstract 3
- 238000006467 substitution reaction Methods 0.000 title abstract 2
- 102000006890 Methyl-CpG-Binding Protein 2 Human genes 0.000 claims abstract 16
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 claims abstract 16
- 230000026683 transduction Effects 0.000 claims abstract 8
- 238000010361 transduction Methods 0.000 claims abstract 8
- 208000029726 Neurodevelopmental disease Diseases 0.000 claims abstract 6
- 239000008194 pharmaceutical composition Substances 0.000 claims abstract 5
- 238000000034 method Methods 0.000 claims abstract 4
- 208000006289 Rett Syndrome Diseases 0.000 claims abstract 3
- 208000015122 neurodegenerative disease Diseases 0.000 claims abstract 3
- 230000000626 neurodegenerative effect Effects 0.000 claims abstract 3
- 230000002265 prevention Effects 0.000 claims abstract 2
- 239000012634 fragment Substances 0.000 claims 16
- 229920001184 polypeptide Polymers 0.000 claims 15
- 102000004196 processed proteins & peptides Human genes 0.000 claims 15
- 108090000765 processed proteins & peptides Proteins 0.000 claims 15
- 241000124008 Mammalia Species 0.000 claims 2
- 101710149951 Protein Tat Proteins 0.000 claims 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- 238000003556 assay Methods 0.000 claims 2
- 230000004071 biological effect Effects 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 238000013518 transcription Methods 0.000 claims 2
- 230000035897 transcription Effects 0.000 claims 2
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims 1
- 102000001708 Protein Isoforms Human genes 0.000 claims 1
- 108010029485 Protein Isoforms Proteins 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 230000002103 transcriptional effect Effects 0.000 claims 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 4
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 108091005461 Nucleic proteins Proteins 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000004927 fusion Effects 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
Abstract
Polipeptid, koji sadrži (i) protein MeCP2 ili biološki aktivni fragment ili derivat spomenutog proteina ili fragmenta, i (ii) domenu transdukcije proteina, pri čemu je protein MeCP2 sjedinjen s domenom transdukcije proteina, naznačen time, da se upotrebljava u postupku liječenja neurodegenerativne ili neurorazvijajuće bolesti; pri čemu spomenuti fragment sadrži najmanje 150 amino kiselina proteina MeCP2; i pri čemu spomenuti derivat spomenutog proteina ili fragmenta ima najmanje 60% identičnosti sa spomenutim proteinom MeCP2 ili njegovim fragmentom; i pri čemu spomenuti fragment ili spomenuti derivat ima najmanje 60% spomenute biološke aktivnosti, kako je određeno transkripcijskim ispitivanjem in vitro opisanim prema Nan, X et al., Cell 88: 471-481 (1997). Patent sadrži još 12 patentnih zahtjeva.
Claims (13)
1. Polipeptid, koji sadrži (i) protein MeCP2 ili biološki aktivni fragment ili derivat spomenutog proteina ili fragmenta, i (ii) domenu transdukcije proteina, pri čemu je protein MeCP2 sjedinjen s domenom transdukcije proteina, naznačen time, da se upotrebljava u postupku liječenja neurodegenerativne ili neurorazvijajuće bolesti; pri čemu spomenuti fragment sadrži najmanje 150 amino kiselina proteina MeCP2; i pri čemu spomenuti derivat spomenutog proteina ili fragmenta ima najmanje 60% identičnosti sa spomenutim proteinom MeCP2 ili njegovim fragmentom; i pri čemu spomenuti fragment ili spomenuti derivat ima najmanje 60% spomenute biološke aktivnosti, kako je određeno transkripcijskim ispitivanjem in vitro opisanim prema Nan, X et al., Cell 88: 471-481 (1997).
2. Polipeptid, koji sadrži (i) protein MeCP2 ili biološki aktivni fragment ili derivat spomenutog proteina ili fragmenta, i (ii) domenu transdukcije proteina, pri čemu je protein MeCP2 sjedinjen s domenom transdukcije proteina, naznačen time, da se upotrebljava u postupku liječenja Rett-sindroma; pri čemu spomenuti fragment sadrži najmanje 150 amino kiselina proteina MeCP2; i pri čemu spomenuti derivat spomenutog proteina ili fragmenta ima najmanje 60% identičnosti sa spomenutim proteinom MeCP2 ili njegovim fragmentom; i pri čemu spomenuti fragment ili spomenuti derivat ima najmanje 60% spomenute biološke aktivnosti, kako je određeno transkripcijskim ispitivanjem in vitro opisanim prema Nan, X et al., Cell 88: 471-481 (1997).
3. Polipeptid prema zahtjevu 1, naznačen time, da se neurorazvijajuća bolest očekuje uslijed redukcije ekspresije MeCP2 ili uslijed oslabljene funkcije MeCP2.
4. Polipeptid prema bilo kojem zahtjevu 1 do 3, naznačen time, da je domena transdukcije proteina proizašla iz transkripcijskog pokretača proteina humanog virusa-1-imunodeficijencije (TAT protein).
5. Polipeptid prema bilo kojem zahtjevu 1 do 4, naznačen time, da protein MeCP2 ili njegov biološki aktivni fragment ili njegov derivat je humanog porijekla.
6. Polipeptid prema bilo kojem zahtjevu 1 do 5, naznačen time, da protein MeCP2 ili njegov biološki aktivni fragment ili njegov derivat je humani MeCP2g izoform-e1 ili humani MeCP2g izoform-e2 ili njegov biološki aktivni fragment ili derivat spomenutih izoforma ili spomenutih fragmenata.
7. Polipeptid prema bilo kojem zahtjevu 1 do 6, naznačen time, da spomenuta domena transdukcije proteina ima najmanje 60%, posebno 70% identičnosti sekvence sa sekvencom amino kiseline od domene transdukcije TAT proteina (SEQ ID NO.54).
8. Polipeptid prema bilo kojem zahtjevu 1 do 7, naznačen time, da liječenje obuhvaća davanje polipeptida sisavcima.
9. Polipeptid prema bilo kojem zahtjevu 1 do 8, naznačen time, da je spomenuti polipeptid obuhvaćen u farmaceutskom sastavu.
10. Polipeptid prema bilo kojem zahtjevu 1 do 9, naznačen time, da se spomenuti farmaceutski sastav daje u dozi od 0,1-200 μg polipeptida po gramu mase tijela sisavca.
11. Polipeptid prema bilo kojem zahtjevu 1 do 10, naznačen time, da se spomenuti farmaceutski sastav daje najmanje jednom dnevno ili svaki drugi dan.
12. Uporaba polipeptida prema bilo kojem zahtjevu 1 do 11, naznačena time, da se koristi za proizvodnju farmaceutskog sastava za prevenciju i/ili liječenje neurodegenerativne ili neurorazvijajuće bolesti.
13. Uporaba prema zahtjevu 12, naznačena time, da je neurorazvijajuća bolest očekivana uslijed redukcije ekspresije MeCP2 ili uslijed oslabljene funkcije MeCP2, posebno kada je neurorazvijajuća bolest Rett-sindrom.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/EP2006/003203 WO2007115578A1 (en) | 2006-04-07 | 2006-04-07 | Synthetic mecp2 sequence for protein substitution therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20110386T1 true HRP20110386T1 (hr) | 2011-08-31 |
Family
ID=36589309
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HR20110386T HRP20110386T1 (hr) | 2006-04-07 | 2011-05-24 | Sintetička sekvenca mecp2 za zamjensku proteinsku terapiju |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US8226930B2 (hr) |
| EP (1) | EP2010563B1 (hr) |
| JP (1) | JP5102283B2 (hr) |
| CN (1) | CN101415726B (hr) |
| AT (1) | ATE500265T1 (hr) |
| AU (1) | AU2006341726B2 (hr) |
| CA (1) | CA2647125C (hr) |
| DE (1) | DE602006020501D1 (hr) |
| DK (1) | DK2010563T3 (hr) |
| ES (1) | ES2362195T3 (hr) |
| HK (1) | HK1121468A1 (hr) |
| HR (1) | HRP20110386T1 (hr) |
| IL (1) | IL194274A (hr) |
| PL (1) | PL2010563T3 (hr) |
| SI (1) | SI2010563T1 (hr) |
| WO (1) | WO2007115578A1 (hr) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IT1398191B1 (it) * | 2010-02-17 | 2013-02-14 | Ist Superiore Sanita | Uso di tossine che attivano le rho gtpasi per il trattamento e/o la prevenzione della sintomatologia associata alla sindrome di rett (rtt). |
| JP6336755B2 (ja) | 2010-11-12 | 2018-06-06 | ザ ジェネラル ホスピタル コーポレイション | ポリコームに関連する非コードｒｎａ |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| WO2013173608A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating mecp2 expression |
| US10174323B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating ATP2A2 expression |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 |
| AU2013262656A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating UTRN expression |
| AP2014008100A0 (en) | 2012-05-16 | 2014-12-31 | Gen Hospital Corp | Compositions and methods for modulating hemoglobingene family expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| WO2014130408A1 (en) * | 2013-02-20 | 2014-08-28 | The Research Foundation For The State University Of New York | Regulators of rab5 activity |
| CA2966044A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| WO2016149455A2 (en) | 2015-03-17 | 2016-09-22 | The General Hospital Corporation | The rna interactome of polycomb repressive complex 1 (prc1) |
| TWI601821B (zh) * | 2016-04-15 | 2017-10-11 | 中央研究院 | 治療神經發展性疾病 |
| US11969479B2 (en) * | 2017-03-24 | 2024-04-30 | The University Court Of The University Of Edinburgh | MeCP2 expression cassettes |
| US11629342B2 (en) | 2017-10-17 | 2023-04-18 | President And Fellows Of Harvard College | Cas9-based transcription modulation systems |
| KR102052097B1 (ko) * | 2017-11-30 | 2019-12-05 | 한국과학기술연구원 | Mecp2 단백질 또는 이를 암호화하는 폴리뉴클레오티드를 포함하는 퇴행성 뇌질환 예방 또는 치료용 조성물 |
| JP2022530095A (ja) * | 2019-04-24 | 2022-06-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | レット症候群の治療に有用な組成物 |
| WO2023235648A1 (en) * | 2022-06-03 | 2023-12-07 | The Regents Of The University Of California | Nuclear Delivery and Transcriptional Repression with a Cell-penetrant MeCP2 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1157275A4 (en) | 1999-02-28 | 2003-01-15 | Univ Washington | NEW TRANSDUCTION MOLECULES AND METHOD FOR USING THE SAME |
| US6709817B1 (en) | 1999-09-07 | 2004-03-23 | Baylor College Of Medicine | Method of screening Rett syndrome by detecting a mutation in MECP2 |
| US20040126877A1 (en) * | 2001-04-20 | 2004-07-01 | Man-Wook Hur | Repressors for hiv transcription and methods thereof |
| JP5830213B2 (ja) * | 2004-02-17 | 2015-12-09 | ザ ホスピタル フォー シック チルドレン | Ｍｅｃｐ２ｅ１遺伝子 |
-
2006
- 2006-04-07 DK DK06724141.4T patent/DK2010563T3/da active
- 2006-04-07 PL PL06724141T patent/PL2010563T3/pl unknown
- 2006-04-07 CN CN2006800541487A patent/CN101415726B/zh not_active Expired - Fee Related
- 2006-04-07 EP EP06724141A patent/EP2010563B1/en active Active
- 2006-04-07 WO PCT/EP2006/003203 patent/WO2007115578A1/en active Application Filing
- 2006-04-07 ES ES06724141T patent/ES2362195T3/es active Active
- 2006-04-07 DE DE602006020501T patent/DE602006020501D1/de active Active
- 2006-04-07 SI SI200631023T patent/SI2010563T1/sl unknown
- 2006-04-07 AT AT06724141T patent/ATE500265T1/de active
- 2006-04-07 CA CA2647125A patent/CA2647125C/en not_active Expired - Fee Related
- 2006-04-07 JP JP2009503417A patent/JP5102283B2/ja not_active Expired - Fee Related
- 2006-04-07 AU AU2006341726A patent/AU2006341726B2/en not_active Ceased
- 2006-04-07 US US12/295,856 patent/US8226930B2/en active Active
-
2008
- 2008-09-22 IL IL194274A patent/IL194274A/en not_active IP Right Cessation
-
2009
- 2009-02-17 HK HK09101479.9A patent/HK1121468A1/xx not_active IP Right Cessation
-
2011
- 2011-05-24 HR HR20110386T patent/HRP20110386T1/hr unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2006341726B2 (en) | 2012-09-20 |
| DE602006020501D1 (de) | 2011-04-14 |
| WO2007115578A8 (en) | 2008-10-30 |
| EP2010563B1 (en) | 2011-03-02 |
| CN101415726B (zh) | 2013-07-31 |
| JP5102283B2 (ja) | 2012-12-19 |
| IL194274A (en) | 2011-07-31 |
| CA2647125A1 (en) | 2007-10-18 |
| CA2647125C (en) | 2015-02-03 |
| DK2010563T3 (da) | 2011-05-23 |
| HK1121468A1 (en) | 2009-04-24 |
| US8226930B2 (en) | 2012-07-24 |
| SI2010563T1 (sl) | 2011-07-29 |
| US20090233856A1 (en) | 2009-09-17 |
| ATE500265T1 (de) | 2011-03-15 |
| WO2007115578A1 (en) | 2007-10-18 |
| JP2009532045A (ja) | 2009-09-10 |
| ES2362195T3 (es) | 2011-06-29 |
| EP2010563A1 (en) | 2009-01-07 |
| AU2006341726A1 (en) | 2007-10-18 |
| CN101415726A (zh) | 2009-04-22 |
| PL2010563T3 (pl) | 2011-11-30 |
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