JP2020517268A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020517268A5 JP2020517268A5 JP2019557373A JP2019557373A JP2020517268A5 JP 2020517268 A5 JP2020517268 A5 JP 2020517268A5 JP 2019557373 A JP2019557373 A JP 2019557373A JP 2019557373 A JP2019557373 A JP 2019557373A JP 2020517268 A5 JP2020517268 A5 JP 2020517268A5
- Authority
- JP
- Japan
- Prior art keywords
- cell
- seq
- cells
- promoter
- subject
- 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.)
- Granted
Links
- 210000004027 cell Anatomy 0.000 claims description 63
- 210000003719 b-lymphocyte Anatomy 0.000 claims description 33
- 108091033319 polynucleotide Proteins 0.000 claims description 25
- 102000040430 polynucleotide Human genes 0.000 claims description 25
- 239000002157 polynucleotide Substances 0.000 claims description 25
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 16
- 230000004069 differentiation Effects 0.000 claims description 10
- 230000035755 proliferation Effects 0.000 claims description 10
- 230000004083 survival effect Effects 0.000 claims description 10
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 8
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 8
- 210000000066 myeloid cell Anatomy 0.000 claims description 8
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 7
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 6
- 150000007523 nucleic acids Chemical group 0.000 claims description 6
- 108060003951 Immunoglobulin Proteins 0.000 claims description 5
- 239000004599 antimicrobial Substances 0.000 claims description 5
- 102000018358 immunoglobulin Human genes 0.000 claims description 5
- 238000009256 replacement therapy Methods 0.000 claims description 5
- 230000001737 promoting effect Effects 0.000 claims description 4
- 108010077544 Chromatin Proteins 0.000 claims description 3
- 210000003483 chromatin Anatomy 0.000 claims description 3
- 102000001714 Agammaglobulinaemia Tyrosine Kinase Human genes 0.000 claims 5
- 108010029445 Agammaglobulinaemia Tyrosine Kinase Proteins 0.000 claims 5
- 206010060880 Monoclonal gammopathy Diseases 0.000 claims 1
- 238000004587 chromatography analysis Methods 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 201000010717 Bruton-type agammaglobulinemia Diseases 0.000 description 22
- 208000016349 X-linked agammaglobulinemia Diseases 0.000 description 22
- 101000864342 Homo sapiens Tyrosine-protein kinase BTK Proteins 0.000 description 20
- 102000016911 Deoxyribonucleases Human genes 0.000 description 16
- 108010053770 Deoxyribonucleases Proteins 0.000 description 16
- 206010020751 Hypersensitivity Diseases 0.000 description 14
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 10
- 239000003623 enhancer Substances 0.000 description 8
- 102000053446 human BTK Human genes 0.000 description 8
- 238000000034 method Methods 0.000 description 8
- 208000024891 symptom Diseases 0.000 description 8
- 108090000623 proteins and genes Proteins 0.000 description 4
- 238000011144 upstream manufacturing Methods 0.000 description 4
- 229920001184 polypeptide Polymers 0.000 description 3
- 108090000765 processed proteins & peptides Proteins 0.000 description 3
- 102000004196 processed proteins & peptides Human genes 0.000 description 3
- 241000282412 Homo Species 0.000 description 2
- 108091092195 Intron Proteins 0.000 description 2
- 239000012472 biological sample Substances 0.000 description 2
- 230000002708 enhancing effect Effects 0.000 description 2
- 230000001629 suppression Effects 0.000 description 2
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024063151A JP7696039B2 (ja) | 2017-04-21 | 2024-04-10 | Ｘｌａ遺伝子療法用の最適化されたレンチウイルスベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762488523P | 2017-04-21 | 2017-04-21 | |
| US62/488,523 | 2017-04-21 | ||
| PCT/US2018/028331 WO2018195297A1 (en) | 2017-04-21 | 2018-04-19 | Optimized lentiviral vector for xla gene therapy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024063151A Division JP7696039B2 (ja) | 2017-04-21 | 2024-04-10 | Ｘｌａ遺伝子療法用の最適化されたレンチウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020517268A JP2020517268A (ja) | 2020-06-18 |
| JP2020517268A5 true JP2020517268A5 (enExample) | 2021-06-10 |
| JP7471821B2 JP7471821B2 (ja) | 2024-04-22 |
Family
ID=63856377
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019557373A Active JP7471821B2 (ja) | 2017-04-21 | 2018-04-19 | Ｘｌａ遺伝子療法用の最適化されたレンチウイルスベクター |
| JP2024063151A Active JP7696039B2 (ja) | 2017-04-21 | 2024-04-10 | Ｘｌａ遺伝子療法用の最適化されたレンチウイルスベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024063151A Active JP7696039B2 (ja) | 2017-04-21 | 2024-04-10 | Ｘｌａ遺伝子療法用の最適化されたレンチウイルスベクター |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20200325458A1 (enExample) |
| EP (1) | EP3612238A4 (enExample) |
| JP (2) | JP7471821B2 (enExample) |
| CN (1) | CN110809627A (enExample) |
| AU (1) | AU2018256412B2 (enExample) |
| WO (1) | WO2018195297A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019232251A1 (en) | 2018-05-30 | 2019-12-05 | The Regents Of The University Of California | Gene editing of monogenic disorders in human hematopoietic stem cells -- correction of x-linked hyper-igm syndrome (xhim) |
| SG11202101594WA (en) | 2018-08-24 | 2021-03-30 | Csl Behring Gene Therapy Inc | Vector production in serum free media |
| US11464872B2 (en) * | 2020-12-07 | 2022-10-11 | Noga Therapeutics Ltd. | Lentiviral vectors for therapeutic expression of BTK in haematopoietic cells |
| WO2025215591A1 (en) | 2024-04-12 | 2025-10-16 | Csl Behring L.L.C. | Modified vectors for xla gene therapy |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6974667B2 (en) * | 2000-06-14 | 2005-12-13 | Gene Logic, Inc. | Gene expression profiles in liver cancer |
| US20160004814A1 (en) * | 2012-09-05 | 2016-01-07 | University Of Washington Through Its Center For Commercialization | Methods and compositions related to regulation of nucleic acids |
-
2018
- 2018-04-19 CN CN201880043435.0A patent/CN110809627A/zh active Pending
- 2018-04-19 EP EP18788504.1A patent/EP3612238A4/en active Pending
- 2018-04-19 JP JP2019557373A patent/JP7471821B2/ja active Active
- 2018-04-19 WO PCT/US2018/028331 patent/WO2018195297A1/en not_active Ceased
- 2018-04-19 US US16/605,740 patent/US20200325458A1/en active Pending
- 2018-04-19 AU AU2018256412A patent/AU2018256412B2/en active Active
-
2024
- 2024-04-10 JP JP2024063151A patent/JP7696039B2/ja active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7592332B2 (ja) | 横隔膜特異的核酸調節エレメントならびにその方法および使用 | |
| US20200407746A1 (en) | Muscle-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof | |
| JP2020517268A5 (enExample) | ||
| JP2020040969A5 (enExample) | ||
| Grine et al. | Dual inhibition of TNFR1 and IFNAR1 in imiquimod-induced psoriasiform skin inflammation in mice | |
| US12162915B2 (en) | Cell penetrating peptides that inhibit IRF5 nuclear localization | |
| JP2019523648A5 (enExample) | ||
| RU2019100525A (ru) | Векторная система на основе аденоассоциированного вируса | |
| Xiao et al. | Therapeutic effects of neuregulin-1 gene transduction in rats with myocardial infarction | |
| EP3551649B1 (en) | Hmgb1 mutants | |
| JPWO2019156137A5 (enExample) | ||
| JP2019517815A (ja) | 細胞傷害性免疫細胞による攻撃に対してがん細胞を増感させるためのｎｋｇ２ｄ活性化リガンドタンパク質の発現 | |
| CN110420331B (zh) | Alkbh5抑制物在治疗病毒感染性疾病中的应用 | |
| Ueda et al. | Deficiency of CRTH2, a prostaglandin D2 receptor, aggravates bleomycin-induced pulmonary inflammation and fibrosis | |
| JP2020505317A5 (enExample) | ||
| JP2018509154A5 (enExample) | ||
| Dey et al. | The role of neutrophils in measles virus–mediated oncolysis differs between B-cell malignancies and is not always enhanced by GCSF | |
| JP2022500353A (ja) | Ｉｇｆ−１−暗号化ｄｎａ作製物及びｈｇｆ−暗号化ｄｎａ作製物を用いた神経病症治療 | |
| Yalvac et al. | VIP-expressing dendritic cells protect against spontaneous autoimmune peripheral polyneuropathy | |
| JP2011529882A5 (enExample) | ||
| Zhang et al. | Avian influenza virus infection induces differential expression of genes in chicken kidney | |
| WO2016112849A1 (zh) | I型干扰素及间充质干细胞在制备抗肿瘤药物中的新用途 | |
| Du et al. | Effect of MSTN propeptide protein on the growth and development of Altay lamb muscle | |
| Mehrabi Habibabadi et al. | Overexpression of Nrf2 in umbilical cord-derived mesenchymal stem cells upregulating cytoprotective genes, TXNRD1 and GCLC | |
| US20240124541A1 (en) | Compositions and methods for treating obesity |