BR112017017810A2 - materiais e métodos para tratamento de hemoglobinopatias - Google Patents

materiais e métodos para tratamento de hemoglobinopatias

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Publication number
BR112017017810A2
BR112017017810A2 BR112017017810A BR112017017810A BR112017017810A2 BR 112017017810 A2 BR112017017810 A2 BR 112017017810A2 BR 112017017810 A BR112017017810 A BR 112017017810A BR 112017017810 A BR112017017810 A BR 112017017810A BR 112017017810 A2 BR112017017810 A2 BR 112017017810A2
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methods
hemoglobinopathies
materials
cells
hbf
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BR112017017810A
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English (en)
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Matthew Hebden Porteus
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Crispr Therapeutics Ag
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Publication of BR112017017810A2 publication Critical patent/BR112017017810A2/pt

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Abstract

a presente invenção refere-se a materiais e métodos para o tratamento de hemoglobinopatias. mais especificamente, o pedido proporciona métodos para produzir células progenitoras que são geneticamente modificadas via edição de genoma para aumentar a produção de hemoglobina fetal (hbf), bem como células progenitoras modificadas (incluindo, por exemplo, cd34 + células tronco hematopoiéticas humanas) que produzem níveis aumentados de hbf, e métodos para usar tais células para o tratamento de hemoglobinopatias tais como anemia falciforme e ß-talassemia.
BR112017017810A 2015-02-23 2016-02-23 materiais e métodos para tratamento de hemoglobinopatias BR112017017810A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562119754P 2015-02-23 2015-02-23
PCT/IB2016/000276 WO2016135557A2 (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies

Publications (1)

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BR112017017810A2 true BR112017017810A2 (pt) 2018-04-10

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BR112017017812A BR112017017812A2 (pt) 2015-02-23 2016-02-23 materiais e métodos para tratamento de hemoglobinopatias
BR112017017810A BR112017017810A2 (pt) 2015-02-23 2016-02-23 materiais e métodos para tratamento de hemoglobinopatias

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US (3) US10738305B2 (pt)
EP (2) EP3262172A2 (pt)
CN (2) CN107532182A (pt)
AU (2) AU2016225179C1 (pt)
BR (2) BR112017017812A2 (pt)
CA (2) CA2977447A1 (pt)
SG (2) SG11201706767RA (pt)
WO (2) WO2016135558A2 (pt)

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