CN110168085A - 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病 - Google Patents

用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病 Download PDF

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Publication number
CN110168085A
CN110168085A CN201880004477.3A CN201880004477A CN110168085A CN 110168085 A CN110168085 A CN 110168085A CN 201880004477 A CN201880004477 A CN 201880004477A CN 110168085 A CN110168085 A CN 110168085A
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galc
subject
nucleic acid
gly
leu
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CN201880004477.3A
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Chinese (zh)
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P·绍博尔奇
M·L·埃斯科拉
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University of Pittsburgh
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University of Pittsburgh
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Priority to CN202410102735.9A priority Critical patent/CN118078860A/zh
Publication of CN110168085A publication Critical patent/CN110168085A/zh
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    • A—HUMAN NECESSITIES
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/48—Reproductive organs
    • A61K35/51—Umbilical cord; Umbilical cord blood; Umbilical stem cells
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    • A61K31/13—Amines
    • A61K31/155—Amidines (), e.g. guanidine (H2N—C(=NH)—NH2), isourea (N=C(OH)—NH2), isothiourea (—N=C(SH)—NH2)
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    • A61K31/255—Esters, e.g. nitroglycerine, selenocyanates of sulfoxy acids or sulfur analogues thereof
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    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
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    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
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CN201880004477.3A 2017-01-20 2018-01-19 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病 Pending CN110168085A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202410102735.9A CN118078860A (zh) 2017-01-20 2018-01-19 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762448433P 2017-01-20 2017-01-20
US62/448,433 2017-01-20
PCT/US2018/014370 WO2018136710A1 (en) 2017-01-20 2018-01-19 Treatment of krabbe disease with umbilical cord blood transplantion (ucbt) and increased galactocerebrosidase (galc) expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202410102735.9A Division CN118078860A (zh) 2017-01-20 2018-01-19 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病

Publications (1)

Publication Number Publication Date
CN110168085A true CN110168085A (zh) 2019-08-23

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CN202410102735.9A Pending CN118078860A (zh) 2017-01-20 2018-01-19 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病
CN201880004477.3A Pending CN110168085A (zh) 2017-01-20 2018-01-19 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病

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CN202410102735.9A Pending CN118078860A (zh) 2017-01-20 2018-01-19 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病

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US (1) US20190336540A1 (enExample)
EP (1) EP3571298A4 (enExample)
JP (2) JP2020505317A (enExample)
KR (1) KR20190106990A (enExample)
CN (2) CN118078860A (enExample)
AU (1) AU2018210326B9 (enExample)
CA (1) CA3042467A1 (enExample)
IL (1) IL266287A (enExample)
WO (1) WO2018136710A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3884057A4 (en) * 2018-12-21 2022-09-07 The University of North Carolina at Chapel Hill OPTIMIZED GALC GENES AND EXPRESSION CASSETTE AND THEIR USE
EP3931337A1 (en) * 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Compositions useful in treatment of krabbe disease
AU2020245415B2 (en) * 2019-03-22 2025-06-26 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders
BR112022021786A2 (pt) * 2020-05-12 2023-01-17 Univ Pennsylvania Composições úteis em tratamento de doença de krabbe
WO2021262498A1 (en) * 2020-06-26 2021-12-30 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods and materials for treating non-malignant disorders or diseases with cord blood

Citations (1)

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Publication number Priority date Publication date Assignee Title
CN104735976A (zh) * 2012-08-07 2015-06-24 Ace生物科学公司 克拉伯病的动物模型

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US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
CN104735976A (zh) * 2012-08-07 2015-06-24 Ace生物科学公司 克拉伯病的动物模型

Non-Patent Citations (4)

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Title
MARIA L ESCOLAR等: "Transplantation of umbilical-cord blood in babies with infantile Krabbe"s disease", 《N ENGL J MED》 *
MOHAMMAD A RAFI等: "Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice", 《MOLECULAR THERAPY》 *
SHAO YH等: "Homo sapiens galactosylceramidase (GALC), transcript variant 1, mRNA，NM_000153.3", 《GENBANK》 *
田晖等: "《新生儿常见病防治与脐带血应用》", 31 May 2016, 金盾出版社 *

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IL266287A (en) 2019-06-30
AU2018210326B9 (en) 2024-04-04
AU2018210326B2 (en) 2024-03-07
AU2018210326A1 (en) 2019-05-23
US20190336540A1 (en) 2019-11-07
JP2023041950A (ja) 2023-03-24
EP3571298A1 (en) 2019-11-27
EP3571298A4 (en) 2020-10-21
CN118078860A (zh) 2024-05-28
KR20190106990A (ko) 2019-09-18
CA3042467A1 (en) 2018-07-26
JP2020505317A (ja) 2020-02-20
WO2018136710A1 (en) 2018-07-26

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