CN110035764B - 卡纳万病的治疗 - Google Patents

卡纳万病的治疗 Download PDF

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Publication number
CN110035764B
CN110035764B CN201780051943.9A CN201780051943A CN110035764B CN 110035764 B CN110035764 B CN 110035764B CN 201780051943 A CN201780051943 A CN 201780051943A CN 110035764 B CN110035764 B CN 110035764B
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aspa
cells
gene
ipscs
npcs
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CN110035764A (zh
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施艳红
晁建飞
李文东
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City of Hope
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City of Hope
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    • A—HUMAN NECESSITIES
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    • 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
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
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    • A01K2217/00—Genetically modified animals
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    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/13—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from connective tissue cells, from mesenchymal cells
    • C12N2506/1307—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from connective tissue cells, from mesenchymal cells from adult fibroblasts
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    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
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    • C12N2740/10011—Retroviridae
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    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Neurosurgery (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Materials For Medical Uses (AREA)
CN201780051943.9A 2016-06-22 2017-06-22 卡纳万病的治疗 Expired - Fee Related CN110035764B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662353515P 2016-06-22 2016-06-22
US62/353,515 2016-06-22
PCT/US2017/038853 WO2017223373A1 (en) 2016-06-22 2017-06-22 Treatment of canavan disease

Publications (2)

Publication Number Publication Date
CN110035764A CN110035764A (zh) 2019-07-19
CN110035764B true CN110035764B (zh) 2023-08-15

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CN201780051943.9A Expired - Fee Related CN110035764B (zh) 2016-06-22 2017-06-22 卡纳万病的治疗

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US (2) US20190307808A1 (https=)
EP (1) EP3474876A4 (https=)
JP (2) JP2019518775A (https=)
CN (1) CN110035764B (https=)
AU (1) AU2017281374A1 (https=)
CA (1) CA3028714A1 (https=)
WO (1) WO2017223373A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022076206A1 (en) * 2020-10-05 2022-04-14 City Of Hope Treatment of canavan disease
CN117999363A (zh) * 2021-08-17 2024-05-07 希望之城 用于无细胞的dna表观遗传的胃肠道癌检测以及治疗的组成物和方法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104114705A (zh) * 2012-02-15 2014-10-22 库瑞瓦格有限责任公司 用于增加编码的治疗性蛋白表达的包含或编码组蛋白茎环和聚腺苷酸序列或聚腺苷酸化信号的核酸
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2172963C (en) * 1993-09-29 2007-09-11 Reuben Matalon Aspartoacylase gene, protein, and methods of screening for mutations associated with canavan disease
PL2861733T3 (pl) * 2012-05-30 2021-04-19 Rowan University Terapia metaboliczna stresu oksydacyjnego w mózgu przez ukierunkowany neuronalny katabolizm kwasu n-acetyloasparaginowego
JP6541577B2 (ja) * 2013-02-06 2019-07-10 ユニバーシティー オブ ロチェスター ミエリン障害の治療のための誘導多能性細胞由来オリゴデンドロサイト前駆細胞

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104114705A (zh) * 2012-02-15 2014-10-22 库瑞瓦格有限责任公司 用于增加编码的治疗性蛋白表达的包含或编码组蛋白茎环和聚腺苷酸序列或聚腺苷酸化信号的核酸
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CD133/CD140a-Based Isolation of Distinct Human Multipotent Neural Progenitor Cells and Oligodendrocyte Progenitor Cells;Jing Wang等;STEM CELLS AND DEVELOPMENT;第22卷(第22期);第1-11页，第9页左栏第3段 *

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Publication number Publication date
US20190307808A1 (en) 2019-10-10
WO2017223373A1 (en) 2017-12-28
JP2023029376A (ja) 2023-03-03
CA3028714A1 (en) 2017-12-28
EP3474876A4 (en) 2020-01-15
EP3474876A1 (en) 2019-05-01
JP2019518775A (ja) 2019-07-04
AU2017281374A1 (en) 2019-01-03
US20240197789A1 (en) 2024-06-20
CN110035764A (zh) 2019-07-19

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