JP2020505317A - 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 - Google Patents

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 Download PDF

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JP2020505317A
JP2020505317A JP2019522742A JP2019522742A JP2020505317A JP 2020505317 A JP2020505317 A JP 2020505317A JP 2019522742 A JP2019522742 A JP 2019522742A JP 2019522742 A JP2019522742 A JP 2019522742A JP 2020505317 A JP2020505317 A JP 2020505317A
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galc
nucleic acid
acid molecule
vector
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JP2020505317A5 (enExample
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マリア ルイーザ エスコラー，
マリア ルイーザ エスコラー，
ポール サボルクス，
ポール サボルクス，
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University of Pittsburgh
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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
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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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JP2019522742A 2017-01-20 2018-01-19 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置 Withdrawn JP2020505317A (ja)

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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

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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

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JP2020505317A5 JP2020505317A5 (enExample) 2021-02-25

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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

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US (2) US12599636B2 (enExample)
EP (1) EP3571298B1 (enExample)
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KR (1) KR20190106990A (enExample)
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AU (1) AU2018210326B9 (enExample)
CA (1) CA3042467A1 (enExample)
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JP2023526311A (ja) * 2020-05-12 2023-06-21 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クラッベ病の治療に有用な組成物
JP2024508620A (ja) * 2021-02-05 2024-02-28 シギロン セラピューティクス， インコーポレイテッド Ｍｐｓ ｖｉ疾患を治療するための組成物、デバイス及び方法

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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
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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

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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023526311A (ja) * 2020-05-12 2023-06-21 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クラッベ病の治療に有用な組成物
JP7832898B2 (ja) 2020-05-12 2026-03-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クラッベ病の治療に有用な組成物
JP2024508620A (ja) * 2021-02-05 2024-02-28 シギロン セラピューティクス， インコーポレイテッド Ｍｐｓ ｖｉ疾患を治療するための組成物、デバイス及び方法

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