In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation (Record no. 50965)

MARC details
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fixed length control field 02648nam a2200469Ia 4500
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control field MX-MdCICY
005 - DATE AND TIME OF LATEST TRANSACTION
control field 20250625160158.0
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Transcribing agency CICY
090 ## - LOCALLY ASSIGNED LC-TYPE CALL NUMBER (OCLC); LOCAL CALL NUMBER (RLIN)
Classification number (OCLC) (R) ; Classification number, CALL (RLIN) (NR) B-16798
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245 10 - TITLE STATEMENT
Title In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation
490 0# - SERIES STATEMENT
Volume/sequential designation Cell, 171(7), p.1495-1507.e15, 2017
520 3# - SUMMARY, ETC.
Summary, etc. Current genome-editing systems generally rely on inducing DNA double-strand breaks (DSBs). This may limit their utility in clinical therapies, as unwanted mutations caused by DSBs can have deleterious effects. CRISPR/Cas9 system has recently been repurposed to enable target gene activation, allowing regulation of endogenous gene expression without creating DSBs. However, in vivo implementation of this gain-of-function system has proven difficult. Here, we report a robust system for in vivo activation of endogenous target genes through trans-epigenetic remodeling. The system relies on recruitment of Cas9 and transcriptional activation complexes to target loci by modified single guide RNAs. As proof-of-concept, we used this technology to treat mouse models of diabetes, muscular dystrophy, and acute kidney disease. Results demonstrate that CRISPR/Cas9-mediated target gene activation can be achieved in vivo, leading to measurable phenotypes and amelioration of disease symptoms. This establishes new avenues for developing targeted epigenetic therapies against human diseases. Video Abstract In vivo delivery of a Cas9-based epigenetic gene activation system ameliorates disease phenotypes in mouse models of type I diabetes, acute kidney injury, and muscular dystrophy
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element CHROMATIN REMODELING
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element CRISPR/CAS9
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element DISEASE MODEL
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element EPIGENETIC MODIFICATION
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element EPIGENETIC THERAPY
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element GENE EDITING
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element MUSCULAR DYSTROPHY
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element REGENERATIVE MEDICINE
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element STEM CELLS
650 14 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name entry element TRANSDIFFERENTIATION
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Liao, H.-K.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Hatanaka, F.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Araoka, T.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Reddy, P.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Wu, M.-Z.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Sui, Y.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Yamauchi, T.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Yamauchi, T.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name O'Keefe, D.D.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Núñez-Delicado, E.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Guillen, P.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Campistol, J.M.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Wu, C.-J.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Lu, L.-F.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Esteban, C.R.
700 12 - ADDED ENTRY--PERSONAL NAME
Personal name Izpisua Belmonte, J.C.
856 40 - ELECTRONIC LOCATION AND ACCESS
Uniform Resource Identifier <a href="https://drive.google.com/file/d/1XHJr6DfWyZ7Jly9U3ZTTCWP80qgHSqae/view?usp=drivesdk">https://drive.google.com/file/d/1XHJr6DfWyZ7Jly9U3ZTTCWP80qgHSqae/view?usp=drivesdk</a>
Public note Para ver el documento ingresa a Google con tu cuenta: @cicy.edu.mx
942 ## - ADDED ENTRY ELEMENTS (KOHA)
Source of classification or shelving scheme Clasificación local
Koha item type Documentos solicitados
Holdings
Lost status Source of classification or shelving scheme Damaged status Not for loan Collection Home library Current library Shelving location Date acquired Total checkouts Full call number Date last seen Price effective from Koha item type
  Clasificación local     Ref1 CICY CICY Documento préstamo interbibliotecario 25.06.2025   B-16798 25.06.2025 25.06.2025 Documentos solicitados