In vivo neuronal gene editing via CRISPR–Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer’s disease

Correction of a pathogenic gene mutation in human embryos

(2017) Hong Ma et al.   NATURE

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Alzheimer's Disease Neuroimaging Initiative: A decade of progress in Alzheimer's disease

(2015) William H. Thies   Alzheimers & Dementia

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

EPPS rescues hippocampus-dependent cognitive deficits in APP/PS1 mice by disaggregation of amyloid-β oligomers and plaques

(2015) Hye Yun Kim et al.   Nature Communications

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Single App knock-in mouse models of Alzheimer's disease

(2014) Takashi Saito et al.   NATURE NEUROSCIENCE

Neuropathological Alterations in Alzheimer Disease

(2013) A. Serrano-Pozo et al.   Cold Spring Harbor Perspectives in Medicine

Role of APP and Aβ in Synaptic Physiology

(2012) Zilai Wang et al.   Current Alzheimer Research

BACE1 Protein Endocytosis and Trafficking Are Differentially Regulated by Ubiquitination at Lysine 501 and the Di-leucine Motif in the Carboxyl Terminus

(2012) Eugene L. Kang et al.   JOURNAL OF BIOLOGICAL CHEMISTRY