Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial Hypercholesterolemia (English)
- New search for: Turunen, Tytteli A K
- New search for: Turunen, Tytteli A K
- New search for: Kurkipuro, Jere
- New search for: Heikura, Tommi
- New search for: Vuorio, Taina
- New search for: Hytönen, Elisa
- New search for: Izsvák, Zsuzsanna
- New search for: Ylä-Herttuala, Seppo
In:
Molecular therapy
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24
, 3
; 620
;
2016
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ISSN:
- Article (Journal) / Print
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Title:Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial Hypercholesterolemia
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Contributors:Turunen, Tytteli A K ( author ) / Kurkipuro, Jere / Heikura, Tommi / Vuorio, Taina / Hytönen, Elisa / Izsvák, Zsuzsanna / Ylä-Herttuala, Seppo
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Published in:Molecular therapy ; 24, 3 ; 620
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Publisher:
- New search for: Cell Press
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Place of publication:Cambridge, MA
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Publication date:2016
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ISSN:
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ZDBID:
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DOI:
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Type of media:Article (Journal)
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Type of material:Print
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Language:English
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Keywords:
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Source:
Table of contents – Volume 24, Issue 3
The tables of contents are generated automatically and are based on the data records of the individual contributions available in the index of the TIB portal. The display of the Tables of Contents may therefore be incomplete.
- 407
-
A New Induction to the Gene and Cell Therapy Hall Of Fame: Genome EditingSadelain, Michel et al. | 2016
- 410
-
In This Issue| 2016
- 411
-
Research Highlights| 2016
- 412
-
A Perspective on the State of Genome EditingCarroll, Dana et al. | 2016
- 414
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CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular DystrophyVandenDriessche, Thierry et al. | 2016
- 416
-
A CRISPR Approach for Reactivating Latent HIV-1Lin, Angel et al. | 2016
- 419
-
Patent Law and Genome Engineering: A Short Guide to a Rapidly Changing LandscapeCloney, Ross et al. | 2016
- 422
-
Salient Features of Endonuclease Platforms for Therapeutic Genome EditingCerto, Michael T et al. | 2016
- 430
-
Genome-editing Technologies for Gene and Cell TherapyMaeder, Morgan L et al. | 2016
- 447
-
Engineered Viruses as Genome Editing DevicesChen, Xiaoyu et al. | 2016
- 458
-
Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research ApplicationsGaj, Thomas et al. | 2016
- 465
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Current Progress in Therapeutic Gene Editing for Monogenic DiseasesPrakash, Versha et al. | 2016
- 475
-
Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome EditingLee, Ciaran M et al. | 2016
- 488
-
Potent and Targeted Activation of Latent HIV-1 Using the CRISPR/dCas9 Activator ComplexSaayman, Sheena M et al. | 2016
- 499
-
CRISPR-mediated Activation of Latent HIV-1 ExpressionLimsirichai, Prajit et al. | 2016
- 508
-
Specific Reactivation of Latent HIV-1 by dCas9-SunTag-VP64-mediated Guide RNA Targeting the HIV-1 PromoterJi, Haiyan et al. | 2016
- 522
-
CRISPR-Cas9 Can Inhibit HIV-1 Replication but NHEJ Repair Facilitates Virus EscapeWang, Gang et al. | 2016
- 527
-
CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular DystrophyHimeda, Charis L et al. | 2016
- 536
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Re-expression of Selected Epigenetically Silenced Candidate Tumor Suppressor Genes in Cervical Cancer by TET2-directed DemethylationTerpstra, Martijn M et al. | 2016
- 548
-
Protein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse BrainBailus, Barbara J et al. | 2016
- 556
-
In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis PigmentosaBakondi, Benjamin et al. | 2016
- 564
-
CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx MiceXu, Li et al. | 2016
- 570
-
Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL NucleasesOsborn, Mark J et al. | 2016
- 582
-
Silent IL2RG Gene Editing in Human Pluripotent Stem CellsLi, Li B et al. | 2016
- 592
-
Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4(+) T CellsGogol-Döring, Andreas et al. | 2016
- 607
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Multidimensional Genome-wide Analyses Show Accurate FVIII Integration by ZFN in Primary Human CellsSivalingam, Jaichandran et al. | 2016
- 620
-
Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial HypercholesterolemiaTurunen, Tytteli A K et al. | 2016
- 636
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Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human GenomeMüller, Maximilian et al. | 2016
- 645
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The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian CellsLee, Ciaran M et al. | 2016