KR20190106990A - 제대혈 이식(ucbt) 및 증가된 갈락토세레브로시다아제(galc) 발현을 이용한 크라베병의 치료 - Google Patents

제대혈 이식(ucbt) 및 증가된 갈락토세레브로시다아제(galc) 발현을 이용한 크라베병의 치료 Download PDF

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KR20190106990A
KR20190106990A KR1020197012608A KR20197012608A KR20190106990A KR 20190106990 A KR20190106990 A KR 20190106990A KR 1020197012608 A KR1020197012608 A KR 1020197012608A KR 20197012608 A KR20197012608 A KR 20197012608A KR 20190106990 A KR20190106990 A KR 20190106990A
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galc
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nucleic acid
gly
leu
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마리아 루이사 에스콜라
폴 사볼치
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유니버시티 오브 피츠버그-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션
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    • A—HUMAN NECESSITIES
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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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    • A61K31/435—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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    • A61K31/5375—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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    • A61K31/7076—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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KR1020197012608A 2017-01-20 2018-01-19 제대혈 이식(ucbt) 및 증가된 갈락토세레브로시다아제(galc) 발현을 이용한 크라베병의 치료 Ceased KR20190106990A (ko)

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

Publications (1)

Publication Number Publication Date
KR20190106990A true KR20190106990A (ko) 2019-09-18

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KR1020197012608A Ceased KR20190106990A (ko) 2017-01-20 2018-01-19 제대혈 이식(ucbt) 및 증가된 갈락토세레브로시다아제(galc) 발현을 이용한 크라베병의 치료

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Country Link
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

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
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
US9986722B2 (en) * 2012-08-07 2018-06-05 Chiesi Farmaceutici S.P.A. Animal model of Krabbe's disease

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IL266287A (en) 2019-06-30
AU2018210326B9 (en) 2024-04-04
AU2018210326B2 (en) 2024-03-07
AU2018210326A1 (en) 2019-05-23
CN110168085A (zh) 2019-08-23
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
CA3042467A1 (en) 2018-07-26
JP2020505317A (ja) 2020-02-20
WO2018136710A1 (en) 2018-07-26

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