JP2020505317A5 - - Google Patents

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Publication number
JP2020505317A5
JP2020505317A5 JP2019522742A JP2019522742A JP2020505317A5 JP 2020505317 A5 JP2020505317 A5 JP 2020505317A5 JP 2019522742 A JP2019522742 A JP 2019522742A JP 2019522742 A JP2019522742 A JP 2019522742A JP 2020505317 A5 JP2020505317 A5 JP 2020505317A5
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JP
Japan
Prior art keywords
subject
nucleic acid
acid molecule
administering
combination according
Prior art date
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Application number
JP2019522742A
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English (en)
Japanese (ja)
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JP2020505317A (ja
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Publication date
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Priority claimed from PCT/US2018/014370 external-priority patent/WO2018136710A1/en
Publication of JP2020505317A publication Critical patent/JP2020505317A/ja
Publication of JP2020505317A5 publication Critical patent/JP2020505317A5/ja
Priority to JP2023016007A priority Critical patent/JP2023041950A/ja
Withdrawn legal-status Critical Current

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JP2019522742A 2017-01-20 2018-01-19 臍帯血移植(ucbt)および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置 Withdrawn JP2020505317A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023016007A JP2023041950A (ja) 2017-01-20 2023-02-06 臍帯血移植(ucbt)および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

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
JP2023016007A Division JP2023041950A (ja) 2017-01-20 2023-02-06 臍帯血移植(ucbt)および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

Publications (2)

Publication Number Publication Date
JP2020505317A JP2020505317A (ja) 2020-02-20
JP2020505317A5 true JP2020505317A5 (enExample) 2021-02-25

Family

ID=62908818

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019522742A Withdrawn JP2020505317A (ja) 2017-01-20 2018-01-19 臍帯血移植(ucbt)および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置
JP2023016007A Pending JP2023041950A (ja) 2017-01-20 2023-02-06 臍帯血移植(ucbt)および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023016007A Pending JP2023041950A (ja) 2017-01-20 2023-02-06 臍帯血移植(ucbt)および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

Country Status (9)

Country Link
US (2) US12599636B2 (enExample)
EP (1) EP3571298B1 (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 (6)

* 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
EA202192328A1 (ru) * 2019-03-22 2022-02-21 Раш Юниверсити Медикал Сентер Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления
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
WO2022170098A2 (en) * 2021-02-05 2022-08-11 Sigilon Therapeutics, Inc. Compositions, devices and methods for treating mps vi disease

Family Cites Families (5)

* 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
US6730297B1 (en) 1999-05-28 2004-05-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing lysosomal storage disorders
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
WO2004042033A2 (en) 2002-11-01 2004-05-21 The Board Of Trustees Of The Leland Stanford Junior University Circulating stem cells and uses related thereto
US9986722B2 (en) * 2012-08-07 2018-06-05 Chiesi Farmaceutici S.P.A. Animal model of Krabbe's disease

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