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MX2019006157A - Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. - Google Patents

Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1.

Info

Publication number
MX2019006157A
MX2019006157A MX2019006157A MX2019006157A MX2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A
Authority
MX
Mexico
Prior art keywords
editing
myo
dystrophin
mediated
mutations
Prior art date
Application number
MX2019006157A
Other languages
English (en)
Inventor
Zhang Yu
Long Chengzu
BASSEL-DUBY Rhonda
Olson Eric
Original Assignee
Univ Texas
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Publication date
Application filed by Univ Texas filed Critical Univ Texas
Publication of MX2019006157A publication Critical patent/MX2019006157A/es

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Abstract

La distrofia muscular de Duchenne (DMD) es una enfermedad ligada a X hereditaria causada por mutaciones en el gen que codifica para distrofina, una proteína requerida para la integridad de la fibra muscular. La divulgación reporta la edición génica mediada por CRISPR/Cpf1 (edición Myo) que es efectiva para corregir la mutación del gen de la distrofina en los ratones mdx, un modelo para la DMD. Además. la divulgación reporta la opbmización de la edición de linea germinal de ratones mdx al diseñar por ingeniería la omisión permanente del exón mutante y al extender la omisión de exón para corregir también la enfermedad a través del suministro pos-natal del virus adenoasociado (AAV). La edición Myo mediada por AAV efectivamente puede rescatar el marco de lectura de la distrofina en ratones mdx, in vivo. La divulgación reporta medios de omisión de exón mediada por la edición Myo que han avanzado exitosamente de tejidos somáticos en ratones a iPSC derivados de pacientes con DMD humanos (células madre plunpotentes inducidas). La edicion myo personalizada se realizo en iF'St: de pacientes con diferentes mutaciones y exitosamente restauró la expresión de la protelna distrofina para todas las mutaciones en cardiomiocitos derivados de iPSC.
MX2019006157A 2016-11-28 2017-11-28 Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. MX2019006157A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662426853P 2016-11-28 2016-11-28
PCT/US2017/063468 WO2018098480A1 (en) 2016-11-28 2017-11-28 Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing

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MX2019006157A true MX2019006157A (es) 2019-11-21

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MX2019006157A MX2019006157A (es) 2016-11-28 2017-11-28 Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1.

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US (1) US20200046854A1 (es)
EP (1) EP3545090A1 (es)
JP (1) JP2019536782A (es)
CN (1) CN110382695A (es)
AU (1) AU2017364106A1 (es)
CA (1) CA3044531A1 (es)
MX (1) MX2019006157A (es)
WO (1) WO2018098480A1 (es)

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