Activating human genes with zinc finger proteins, transcription activator-like effectors and CRISPR/Cas9 for gene therapy and regenerative medicine.

Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector. Nucleic Acids Res (2014) 1.59

Genome-wide specificity of DNA binding, gene regulation, and chromatin remodeling by TALE- and CRISPR/Cas9-based transcriptional activators. Genome Res (2015) 0.92

Lentivirus pre-packed with Cas9 protein for safer gene editing. Gene Ther (2016) 0.80

Prolonged re-expression of the hypermethylated gene EPB41L3 using artificial transcription factors and epigenetic drugs. Epigenetics (2015) 0.80

Optimized tuning of TALEN specificity using non-conventional RVDs. Sci Rep (2015) 0.79

CRISPR/Cas9: molecular tool for gene therapy to target genome and epigenome in the treatment of lung cancer. Cancer Gene Ther (2015) 0.79

CRISPR-on system for the activation of the endogenous human INS gene. Gene Ther (2016) 0.78

Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS. Adv Drug Deliv Rev (2016) 0.78

New approaches to manipulating the epigenome. Dialogues Clin Neurosci (2014) 0.77

Enhanced MyoD-induced transdifferentiation to a myogenic lineage by fusion to a potent transactivation domain. ACS Synth Biol (2014) 0.75

A TALE nuclease architecture for efficient genome editing. Nat Biotechnol (2010) 26.47

Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. Nat Biotechnol (2011) 12.86

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat Biotechnol (2013) 11.35

CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes. Cell (2013) 9.55

RNA-guided gene activation by CRISPR-Cas9-based transcription factors. Nat Methods (2013) 4.85

CRISPR RNA-guided activation of endogenous human genes. Nat Methods (2013) 4.69

Positive and negative regulation of endogenous genes by designed transcription factors. Proc Natl Acad Sci U S A (2000) 3.92

Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system. Cell Res (2013) 3.50

Synergistic and tunable human gene activation by combinations of synthetic transcription factors. Nat Methods (2013) 3.26

Robust, synergistic regulation of human gene expression using TALE activators. Nat Methods (2013) 2.89

Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers. Nucleic Acids Res (2012) 2.83

Induction of angiogenesis in a mouse model using engineered transcription factors. Nat Med (2002) 2.81

Tunable and multifunctional eukaryotic transcription factors based on CRISPR/Cas. ACS Synth Biol (2013) 1.58

Re-activation of a dormant tumor suppressor gene maspin by designed transcription factors. Oncogene (2006) 1.35

Exploring strategies for the design of artificial transcription factors: targeting sites proximal to known regulatory regions for the induction of gamma-globin expression and the treatment of sickle cell disease. J Biol Chem (2004) 1.30

Reprogramming to pluripotency using designer TALE transcription factors targeting enhancers. Stem Cell Reports (2013) 1.22

Design of artificial transcription factors to selectively regulate the pro-apoptotic bax gene. Nucleic Acids Res (2003) 0.97

The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice. Hum Mol Genet (2009) 0.93

Gene transfer of an engineered zinc finger protein enhances the anti-angiogenic defense system. Mol Ther (2007) 0.91

A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins. Mol Ther Nucleic Acids (2013) 0.89

Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients. Mol Ther (2013) 1.63

Pleiotrophin regulates serine phosphorylation and the cellular distribution of beta-adducin through activation of protein kinase C. Proc Natl Acad Sci U S A (2005) 1.10

Fyn is a downstream target of the pleiotrophin/receptor protein tyrosine phosphatase beta/zeta-signaling pathway: regulation of tyrosine phosphorylation of Fyn by pleiotrophin. Biochem Biophys Res Commun (2005) 1.06

Pleiotrophin stimulates tyrosine phosphorylation of beta-adducin through inactivation of the transmembrane receptor protein tyrosine phosphatase beta/zeta. Biochem Biophys Res Commun (2005) 1.05

Secretion of pleiotrophin stimulates breast cancer progression through remodeling of the tumor microenvironment. Proc Natl Acad Sci U S A (2007) 1.00

Value of measuring plasmatic levels of neurosin in the diagnosis of Alzheimer's disease. J Alzheimers Dis (2008) 0.94

Pleiotrophin produced by multiple myeloma induces transdifferentiation of monocytes into vascular endothelial cells: a novel mechanism of tumor-induced vasculogenesis. Blood (2008) 0.94

Identification of the angiogenesis signaling domain in pleiotrophin defines a mechanism of the angiogenic switch. Biochem Biophys Res Commun (2006) 0.93

Scaffold-mediated lentiviral transduction for functional tissue engineering of cartilage. Proc Natl Acad Sci U S A (2014) 0.89

Pleiotrophin expression during odontogenesis. J Histochem Cytochem (2012) 0.80