Development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome

Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome

(2019) Ergin Beyret et al.   NATURE MEDICINE

Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome

(2018) Leslie B. Gordon et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

A novel somatic mutation achieves partial rescue in a child with Hutchinson-Gilford progeria syndrome

(2016) Daniel Z Bar et al.   JOURNAL OF MEDICAL GENETICS

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

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Progeria: A Paradigm for Translational Medicine

(2014) Leslie B. Gordon et al.   CELL

Impact of Farnesylation Inhibitors on Survival in Hutchinson-Gilford Progeria Syndrome

(2014) Leslie B. Gordon et al.   CIRCULATION

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Prelamin A causes progeria through cell-extrinsic mechanisms and prevents cancer invasion

(2013) Jorge de la Rosa et al.   Nature Communications

Splicing-Directed Therapy in a New Mouse Model of Human Accelerated Aging

(2011) F. G. Osorio et al.   Science Translational Medicine

Fast and accurate long-read alignment with Burrows–Wheeler transform

(2010) Heng Li et al.   BIOINFORMATICS

The Sequence Alignment/Map format and SAMtools

(2009) H. Li et al.   BIOINFORMATICS