A TALE nuclease architecture for efficient genome editing

Jeffrey C. Miller, Siyuan Tan, Guijuan Qiao, +17

Nature Biotechnology

Natural SciencesMolecular BiologyDna ReplicationGenetic EngineeringGene Editing +3

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

Kosuke Yusa, S. Tamir Rashid, Hélène Strick‐Marchand, +17

Nature

α1-Antitrypsin DeficiencyInduced Pluripotent Stem CellMedicineGeneticsGene Correction +5

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Hojun Li, Virginia Haurigot, Yannick Doyon, +17

Nature

Vivo GenomeKnockout MouseEngineeringGenome SurgeryGenetics +10

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

Elena Provasi, Pietro Genovese, Angelo Lombardo, +18

Nature Medicine

ChromatinCell TherapyLentiviral Gene TransferMixed-phenotype Acute LeukemiaT-regulatory Cell +8

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Megan D. Hoban, Gregory J. Cost, Matthew Mendel, +22

Blood

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

Russell C. DeKelver, Vivian M. Choi, Erica A. Moehle, +24

Genome Research

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Rajiv P. Sharma, Xavier M. Anguela, Yannick Doyon, +13

Blood

LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson's disease patients: Reversal by gene correction

Laurie H. Sanders, Josée Laganière, Oliver Cooper, +13

Neurobiology of Disease

MitophagyNeurodegenerative DiseasesMolecular NeuroscienceMitochondrial FunctionGenetics +9

Rapid and efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells

Jeffrey B. Doyon, Bryan Zeitler, Jackie Cheng, +13

Nature Cell Biology

Endocytic PathwayGenome-edited Mammalian CellsIntracellular TraffickingCellular BiochemistrySystems Biology +4

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

Bryan Zeitler, Steven Froelich, Kimberly Marlen, +48

Nature Medicine

Transcriptional RegulationNeurodegenerative DiseasesHuntington ’Genetic DisorderMedicine +9