CN118078860A - 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
CN118078860A
CN118078860A CN202410102735.9A CN202410102735A CN118078860A CN 118078860 A CN118078860 A CN 118078860A CN 202410102735 A CN202410102735 A CN 202410102735A CN 118078860 A CN118078860 A CN 118078860A
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galc
nucleic acid
subject
acid molecule
disease
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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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    • 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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    • A61K31/155—Amidines (), e.g. guanidine (H2N—C(=NH)—NH2), isourea (N=C(OH)—NH2), isothiourea (—N=C(SH)—NH2)
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    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/255—Esters, e.g. nitroglycerine, selenocyanates of sulfoxy acids or sulfur analogues thereof
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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—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
    • A61K31/4353—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
    • 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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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • 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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CN202410102735.9A 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 CN118078860A (zh)

Applications Claiming Priority (4)

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
CN201880004477.3A 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

Related Parent Applications (1)

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CN201880004477.3A Division 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

Publications (1)

Publication Number Publication Date
CN118078860A true CN118078860A (zh) 2024-05-28

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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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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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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
PE20212072A1 (es) * 2019-02-26 2021-10-26 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe
US20220152165A1 (en) * 2019-03-22 2022-05-19 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders
AU2021271635A1 (en) * 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
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

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
EP2882284B1 (en) * 2012-08-07 2017-11-15 Chiesi Farmaceutici S.p.A. Animal model of krabbe's disease

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JP2020505317A (ja) 2020-02-20
WO2018136710A1 (en) 2018-07-26
EP3571298A1 (en) 2019-11-27
EP3571298A4 (en) 2020-10-21
JP2023041950A (ja) 2023-03-24
CA3042467A1 (en) 2018-07-26
IL266287A (en) 2019-06-30
CN110168085A (zh) 2019-08-23
AU2018210326B2 (en) 2024-03-07
AU2018210326B9 (en) 2024-04-04
US20190336540A1 (en) 2019-11-07
AU2018210326A1 (en) 2019-05-23
KR20190106990A (ko) 2019-09-18

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