MX2017014665A - Edicion de genes de mutaciones intronicas profundas. - Google Patents

Edicion de genes de mutaciones intronicas profundas.

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Publication number
MX2017014665A
MX2017014665A MX2017014665A MX2017014665A MX2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A
Authority
MX
Mexico
Prior art keywords
crispr
cas
gene editing
deep intronic
intronic mutations
Prior art date
Application number
MX2017014665A
Other languages
English (en)
Inventor
Scaria Abraham
Ruan Guoxiang
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2017014665A publication Critical patent/MX2017014665A/es

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Abstract

Se proporcionan en el presente documento composiciones, métodos, kits, y partículas virales para el tratamiento de una enfermedad o trastorno asociados con una mutación intrónica profunda utilizando un sistema (Cas) asociado a Repeticiones Palindrómicas Cortas Agrupadas Regularmente Interespaciadas (CRISPR, por sus siglas en inglés) - (CRISPR-Cas) modificado genéticamente, no natural. En algunos aspectos, se proporciona en el presente documento un sistema CRISPR-Cas autolimitante.
MX2017014665A 2015-05-16 2016-04-15 Edicion de genes de mutaciones intronicas profundas. MX2017014665A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562162720P 2015-05-16 2015-05-16
PCT/US2016/027987 WO2016186772A2 (en) 2015-05-16 2016-04-15 Gene editing of deep intronic mutations

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MX2017014665A true MX2017014665A (es) 2018-04-24

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MX2017014665A MX2017014665A (es) 2015-05-16 2016-04-15 Edicion de genes de mutaciones intronicas profundas.
MX2023006046A MX2023006046A (es) 2015-05-16 2017-11-15 Edicion de genes de mutaciones intronicas profundas.

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EP (1) EP3298134B8 (es)
JP (2) JP6985250B2 (es)
KR (2) KR102584873B1 (es)
CN (2) CN107849547B (es)
AU (2) AU2016265255B2 (es)
BR (1) BR112017024514A2 (es)
CA (1) CA2986021A1 (es)
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