Development of an isogenic human cell trio that models polyglutamine disease

Biallelic and gene-wide genomic substitution for endogenous intron and retroelement mutagenesis in human cells

(2022) Tomoyuki Ohno et al.   Nature Communications

Use of single guided Cas9 nickase to facilitate precise and efficient genome editing in human iPSCs

(2021) Pan P. Li et al.   Scientific Reports

CRISPR/Cas9 mediated gene correction ameliorates abnormal phenotypes in spinocerebellar ataxia type 3 patient-derived induced pluripotent stem cells

(2021) Lang He et al.   Translational Psychiatry

Generation of New Isogenic Models of Huntington’s Disease Using CRISPR-Cas9 Technology

(2020) Magdalena Dabrowska et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Ropinirole hydrochloride remedy for amyotrophic lateral sclerosis – Protocol for a randomized, double-blind, placebo-controlled, single-center, and open-label continuation phase I/IIa clinical trial (ROPALS trial)

(2019) Satoru Morimoto et al.   Regenerative Therapy

Genome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9

(2018) Ronen Ben Jehuda et al.   Stem Cell Reviews and Reports

Genome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9

(2018) Ronen Ben Jehuda et al.   Stem Cell Reviews and Reports

Polyglutamine Repeats in Neurodegenerative Diseases

(2018) Andrew P. Lieberman et al.   Annual Review of Pathology-Mechanisms of Disease

Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent

(2018) Koki Fujimori et al.   NATURE MEDICINE

Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells

(2017) Xiaohong Xu et al.   Stem Cell Reports

Pluripotent stem cells in disease modelling and drug discovery

(2016) Yishai Avior et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Generation of an isogenic, gene-corrected control cell line of the spinocerebellar ataxia type 2 patient-derived iPSC line H271

(2016) Adele G. Marthaler et al.   Stem Cell Research

Ataxin-2 Regulates RGS8 Translation in a New BAC-SCA2 Transgenic Mouse Model

(2015) Warunee Dansithong et al.   PLoS Genetics

CRISPRdirect: software for designing CRISPR/Cas guide RNA with reduced off-target sites

(2014) Yuki Naito et al.   BIOINFORMATICS

Investigating human disease using stem cell models

(2014) Jared L. Sterneckert et al.   NATURE REVIEWS GENETICS

Genetic Background Drives Transcriptional Variation in Human Induced Pluripotent Stem Cells

(2014) Foad Rouhani et al.   PLoS Genetics

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

The Chicken Hypersensitivity Site 4 Core Insulator Blocks Promoter Interference in Lentiviral Vectors

(2013) Naoya Uchida et al.   Human Gene Therapy Methods

NIH Image to ImageJ: 25 years of image analysis

(2012) Caroline A Schneider et al.   NATURE METHODS

Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations

(2011) Frank Soldner et al.   CELL

Ataxin-2 intermediate-length polyglutamine expansions are associated with increased risk for ALS

(2010) Andrew C. Elden et al.   NATURE

Elite and stochastic models for induced pluripotent stem cell generation

(2009) Shinya Yamanaka   NATURE