JP2011511090A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2011511090A5 JP2011511090A5 JP2010546007A JP2010546007A JP2011511090A5 JP 2011511090 A5 JP2011511090 A5 JP 2011511090A5 JP 2010546007 A JP2010546007 A JP 2010546007A JP 2010546007 A JP2010546007 A JP 2010546007A JP 2011511090 A5 JP2011511090 A5 JP 2011511090A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- cells
- growth factor
- layer
- eye
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Ceased
Links
- 210000004027 cell Anatomy 0.000 claims description 17
- 239000003102 growth factor Substances 0.000 claims description 14
- 239000010410 layer Substances 0.000 claims description 14
- 210000003994 retinal ganglion cell Anatomy 0.000 claims description 14
- 239000013604 expression vector Substances 0.000 claims description 8
- 239000013598 vector Substances 0.000 claims description 8
- 239000013607 AAV vector Substances 0.000 claims description 6
- 108010025020 Nerve Growth Factor Proteins 0.000 claims description 6
- 102000015336 Nerve Growth Factor Human genes 0.000 claims description 6
- 229940053128 nerve growth factor Drugs 0.000 claims description 6
- 108091008695 photoreceptors Proteins 0.000 claims description 6
- 238000003259 recombinant expression Methods 0.000 claims description 6
- 210000001525 retina Anatomy 0.000 claims description 5
- 238000011065 in-situ storage Methods 0.000 claims description 4
- 208000030533 eye disease Diseases 0.000 claims description 3
- 238000002513 implantation Methods 0.000 claims description 3
- 210000004498 neuroglial cell Anatomy 0.000 claims description 3
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims description 2
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims description 2
- 229940127291 Calcium channel antagonist Drugs 0.000 claims description 2
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims description 2
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 claims description 2
- 108091026890 Coding region Proteins 0.000 claims description 2
- 102000034615 Glial cell line-derived neurotrophic factor Human genes 0.000 claims description 2
- 108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 claims description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 2
- 108010067390 Viral Proteins Proteins 0.000 claims description 2
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 2
- 239000000480 calcium channel blocker Substances 0.000 claims description 2
- 239000012792 core layer Substances 0.000 claims description 2
- 230000007850 degeneration Effects 0.000 claims description 2
- 210000002287 horizontal cell Anatomy 0.000 claims description 2
- 208000015181 infectious disease Diseases 0.000 claims description 2
- 238000002347 injection Methods 0.000 claims description 2
- 239000007924 injection Substances 0.000 claims description 2
- 208000002780 macular degeneration Diseases 0.000 claims description 2
- 150000007523 nucleic acids Chemical group 0.000 claims description 2
- 230000003076 paracrine Effects 0.000 claims description 2
- 210000000608 photoreceptor cell Anatomy 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 210000000964 retinal cone photoreceptor cell Anatomy 0.000 claims description 2
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims description 2
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 19
- 208000022873 Ocular disease Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 description 18
- 230000002207 retinal effect Effects 0.000 description 3
- 102000053171 Glial Fibrillary Acidic Human genes 0.000 description 2
- 101710193519 Glial fibrillary acidic protein Proteins 0.000 description 2
- 210000005046 glial fibrillary acidic protein Anatomy 0.000 description 2
- VEEGZPWAAPPXRB-BJMVGYQFSA-N (3e)-3-(1h-imidazol-5-ylmethylidene)-1h-indol-2-one Chemical compound O=C1NC2=CC=CC=C2\C1=C/C1=CN=CN1 VEEGZPWAAPPXRB-BJMVGYQFSA-N 0.000 description 1
- 210000005156 Müller Glia Anatomy 0.000 description 1
- 229940121369 angiogenesis inhibitor Drugs 0.000 description 1
- 239000004037 angiogenesis inhibitor Substances 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 238000002054 transplantation Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US2699008P | 2008-02-07 | 2008-02-07 | |
| US9322808P | 2008-08-29 | 2008-08-29 | |
| PCT/US2009/033275 WO2009100253A1 (en) | 2008-02-07 | 2009-02-05 | Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2011511090A JP2011511090A (ja) | 2011-04-07 |
| JP2011511090A5 true JP2011511090A5 (cg-RX-API-DMAC7.html) | 2013-03-28 |
Family
ID=40939054
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010546007A Ceased JP2011511090A (ja) | 2008-02-07 | 2009-02-05 | 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US8242093B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2247258A4 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2011511090A (cg-RX-API-DMAC7.html) |
| CN (1) | CN101980668A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2009212309B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2713755A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2009100253A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101980668A (zh) * | 2008-02-07 | 2011-02-23 | 赛拉基因公司 | 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器 |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| CN107937341A (zh) * | 2011-02-25 | 2018-04-20 | 国立研究开发法人理化学研究所 | 视网膜色素上皮细胞片层的生产方法 |
| CN105120901A (zh) | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
| US20160256571A1 (en) * | 2013-09-30 | 2016-09-08 | Sanofi | Invention |
| WO2015082690A1 (en) * | 2013-12-06 | 2015-06-11 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject |
| WO2016164920A1 (en) * | 2015-04-09 | 2016-10-13 | Cornell University | Gene therapy to prevent reactions to allergens |
| CA3004807C (en) * | 2015-12-04 | 2022-02-22 | Universite Pierre Et Marie Curie (Paris 6) | Promoters and uses thereof |
| US20190269755A1 (en) * | 2016-04-29 | 2019-09-05 | Gensight Biologics Sa | Optogenetic visual restoration using chrimson |
| PL239642B1 (pl) * | 2018-08-16 | 2021-12-20 | Pomorski Univ Medyczny W Szczecinie | Kompozycja do doszklistkowego podawania białka leczniczego |
| CA3199230A1 (en) * | 2020-10-22 | 2022-04-28 | Visgenx, Inc. | Elovl2 constructs for human gene therapy |
| JP2025540499A (ja) * | 2022-12-05 | 2025-12-11 | ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 色素上皮由来因子ペプチドおよび網膜変性症を処置するための使用 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5932479A (en) | 1988-09-26 | 1999-08-03 | Auburn University | Genetic engineering of plant chloroplasts |
| ATE246935T1 (de) * | 1997-06-11 | 2003-08-15 | Acorda Therapeutics | Zns neuroregenerative zusammensetzungen und deren verwendungen |
| WO2001072959A2 (en) | 2000-03-01 | 2001-10-04 | Auburn University | Plastid transformation vectors for expressing human proteins in plants |
| JP2002534139A (ja) * | 1999-01-05 | 2002-10-15 | マサチューセッツ・アイ・アンド・イア・インファーマリー | 網膜と脈絡膜への経強膜徐放性薬剤標的送達 |
| WO2000054813A2 (en) * | 1999-03-15 | 2000-09-21 | Chiron Corporation | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| US6943153B1 (en) * | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| CA2401855A1 (en) | 2000-02-29 | 2001-09-07 | Auburn University | Genetic engineering of drought tolerance via a plastid genome |
| US7354760B2 (en) | 2001-12-26 | 2008-04-08 | University Of Central Florida Research Foundation, Inc. | Expression of protective antigens in transgenic chloroplasts |
| AU2003253781A1 (en) | 2002-07-03 | 2004-01-23 | University Of Central Florida | Expression of human interferon in transgenic chloroplasts |
| WO2006101634A1 (en) * | 2005-02-17 | 2006-09-28 | The Regents Of The University Of California | Müller cell specific gene therapy |
| US20060188481A1 (en) * | 2005-02-22 | 2006-08-24 | Saitama Medical School | Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder |
| JP2008537478A (ja) * | 2005-02-24 | 2008-09-18 | ザ・スクリプス・リサーチ・インステイチユート | 単離骨髄球様骨髄細胞集団及びそれを用いた治療方法 |
| EP1885173A4 (en) | 2005-05-27 | 2009-02-18 | Univ Central Florida | CHLOROPLASTE DEVELOPED FOR THE EXPRESSION OF PHARMACEUTICAL PROTEINS |
| CN101980668A (zh) * | 2008-02-07 | 2011-02-23 | 赛拉基因公司 | 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器 |
-
2009
- 2009-02-05 CN CN2009801043603A patent/CN101980668A/zh active Pending
- 2009-02-05 EP EP09707354A patent/EP2247258A4/en not_active Withdrawn
- 2009-02-05 CA CA2713755A patent/CA2713755A1/en not_active Abandoned
- 2009-02-05 WO PCT/US2009/033275 patent/WO2009100253A1/en not_active Ceased
- 2009-02-05 AU AU2009212309A patent/AU2009212309B2/en not_active Ceased
- 2009-02-05 JP JP2010546007A patent/JP2011511090A/ja not_active Ceased
- 2009-02-05 US US12/366,596 patent/US8242093B2/en not_active Expired - Fee Related
-
2011
- 2011-08-17 US US13/212,078 patent/US8420619B2/en not_active Expired - Fee Related
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2011511090A5 (cg-RX-API-DMAC7.html) | ||
| JP7182873B2 (ja) | 多重ベクターシステム及びその使用 | |
| RU2018142768A (ru) | Варианты капсидов аденоассоциированного вируса и способы их применения | |
| HRP20211024T1 (hr) | Aav vektori za gensku terapiju mrežnice i cns-a | |
| UA130154C2 (uk) | Спосіб безклітинного одержання днк-вектора з замкненими кінцями | |
| JP2021097675A (ja) | 中枢神経系を標的化したaavベクター | |
| JP2019089787A5 (cg-RX-API-DMAC7.html) | ||
| EP3387137A1 (en) | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) | |
| JP2015523379A5 (cg-RX-API-DMAC7.html) | ||
| HRP20171334T1 (hr) | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe | |
| CN111818942A (zh) | 非病毒dna载体以及其用于产生抗体和融合蛋白的用途 | |
| JP2015501156A5 (cg-RX-API-DMAC7.html) | ||
| MX2013001182A (es) | Acido nucleico que comprende o codifica para una secuencia de tallo-asa de histona y poli (a) o una señal de poliadenilacion para incrementar la expresion de una proteina codificada. | |
| CN113301956B (zh) | 控制器官功能的基因治疗方法 | |
| Hellstrom et al. | Retinal ganglion cell gene therapy and visual system repair | |
| IL308645A (en) | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies | |
| JP7289306B2 (ja) | 網膜障害を治療するための組成物及び方法 | |
| CN112029773B (zh) | 编码bdnf的核酸及其应用 | |
| JP7413629B2 (ja) | Igf-1異型体を発現させるdnaコンストラクトを用いた神経病症の治療 | |
| US12472270B2 (en) | Methods and compositions for reprogramming Müller Glia | |
| CA3228666A1 (en) | Compositions and methods for transgene expression | |
| KR20210130578A (ko) | 폐섬유증 치료 또는 예방을 위한 세포 치료제 조성물 | |
| EA049462B1 (ru) | Способы генной терапии для контроля функции органов | |
| US20210308169A1 (en) | Methods and compositions for treatment of retinal degenerative diseases | |
| RU2021101475A (ru) | Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования |