CA3233534A1 - Direct transdifferentiation for treatment of neurological disease - Google Patents

Direct transdifferentiation for treatment of neurological disease Download PDF

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Publication number
CA3233534A1
CA3233534A1 CA3233534A CA3233534A CA3233534A1 CA 3233534 A1 CA3233534 A1 CA 3233534A1 CA 3233534 A CA3233534 A CA 3233534A CA 3233534 A CA3233534 A CA 3233534A CA 3233534 A1 CA3233534 A1 CA 3233534A1
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rest
cells
protein
neurons
variant
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CA3233534A
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English (en)
French (fr)
Inventor
Haibo Zhou
Xinde Hu
Jinlin SU
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Shanghai Genemagic Biosciences Co Ltd
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Shanghai Genemagic Biosciences Co Ltd
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Publication of CA3233534A1 publication Critical patent/CA3233534A1/en
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    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
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    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61P25/08Antiepileptics; Anticonvulsants
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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N5/06Animal cells or tissues; Human cells or tissues
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CA3233534A 2021-09-30 2022-09-30 Direct transdifferentiation for treatment of neurological disease Pending CA3233534A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN202111158620.4 2021-09-30
CN202111158620.4A CN115887655B (zh) 2021-09-30 2021-09-30 直接转分化治疗神经系统疾病
PCT/CN2022/123409 WO2023051802A1 (zh) 2021-09-30 2022-09-30 直接转分化治疗神经系统疾病

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CA3233534A1 true CA3233534A1 (en) 2023-04-06

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CA3233534A Pending CA3233534A1 (en) 2021-09-30 2022-09-30 Direct transdifferentiation for treatment of neurological disease

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US (1) US20250115650A1 (enExample)
EP (1) EP4410296A4 (enExample)
JP (1) JP2024534667A (enExample)
KR (1) KR20240082366A (enExample)
CN (3) CN115887655B (enExample)
AU (1) AU2022357103A1 (enExample)
CA (1) CA3233534A1 (enExample)
WO (1) WO2023051802A1 (enExample)

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Publication number Priority date Publication date Assignee Title
WO2024199511A1 (zh) * 2023-03-30 2024-10-03 上海鲸奇生物科技有限公司 治疗神经系统疾病的表达载体和组合物
WO2025173411A1 (ja) * 2024-02-14 2025-08-21 国立大学法人筑波大学 網膜神経節細胞分化誘導用キット、医薬組成物及び緑内障モデル

Family Cites Families (10)

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Publication number Priority date Publication date Assignee Title
FR2774698B1 (fr) * 1998-02-12 2002-03-01 Rhone Poulenc Rorer Sa Utilisation d'elements de regulation negative pour l'expression neurospecifique de transgenes
ITVA20060041A1 (it) * 2006-07-05 2008-01-06 Dialectica Srl Uso di composti derivati amminotiazolici, di loro composizioni farmaceutiche, nel trattamento di malattie caratterizzate dalla anormale repressione della trascrizione genica, particolarmente il morbo di huntington
WO2009058014A2 (en) * 2007-11-02 2009-05-07 Vereniging Voor Christelijk Hoger Onderwijs, Wetenschappelijk Onderzoek En Patientenzorg Polypeptides involved in neuronal regeneration-associated gene expression
JP5850321B2 (ja) * 2010-02-10 2016-02-03 公立大学法人横浜市立大学 神経選択的転写抑制因子NRSFに特異的に結合するmSin3Bに結合する化合物の利用
WO2014071157A1 (en) * 2012-11-01 2014-05-08 The Regents Of The University Of California Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases
WO2016019315A2 (en) * 2014-08-01 2016-02-04 Oregon Health & Science University Methods and compositions useful in manipulating the stability of re1 silencing transcription factor
EP3622057A1 (en) * 2017-05-12 2020-03-18 New York Stem Cell Foundation, Inc. Systems
CA3096691A1 (en) * 2018-04-11 2019-10-17 The Regents Of The University Of California Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders
JP7555542B2 (ja) * 2018-06-18 2024-09-25 ユニバーシティー オブ ロチェスター 統合失調症及び他の神経精神障害の治療方法
MX2022006214A (es) * 2019-11-22 2022-06-22 Alcamena Stem Cell Therapeutics Llc Composiciones y métodos para desreprimir genes diana del factor de transcripción silenciador de re1.

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CN118201623A (zh) 2024-06-14
KR20240082366A (ko) 2024-06-10
CN115887655A (zh) 2023-04-04
US20250115650A1 (en) 2025-04-10
CN115887655B (zh) 2025-06-17
EP4410296A1 (en) 2024-08-07
CN120754249A (zh) 2025-10-10
WO2023051802A1 (zh) 2023-04-06
JP2024534667A (ja) 2024-09-20
EP4410296A4 (en) 2025-10-15
AU2022357103A1 (en) 2024-05-02

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