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

Emerging Gene Therapies for Genetic Hearing Loss

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Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c

(2017) Bifeng Pan et al.   NATURE BIOTECHNOLOGY

RNA Interference Prevents Autosomal-Dominant Hearing Loss

(2016) Seiji B. Shibata et al.   AMERICAN JOURNAL OF HUMAN 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

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

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

New treatment options for hearing loss

(2015) Ulrich Müller et al.   NATURE REVIEWS DRUG DISCOVERY

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

(2015) C. Long et al.   SCIENCE

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Transmembrane channel-like (TMC) genes are required for auditory and vestibular mechanosensation

(2014) Yoshiyuki Kawashima et al.   PFLUGERS ARCHIV-EUROPEAN JOURNAL OF PHYSIOLOGY

A Novel DFNA36 Mutation in TMC1 Orthologous to the Beethoven (Bth) Mouse Associated with Autosomal Dominant Hearing Loss in a Chinese Family

(2014) Yali Zhao et al.   PLoS One

Sound Strategies for Hearing Restoration

(2014) G. S. G. Geleoc et al.   SCIENCE

Hair Cell Overexpression of Islet1 Reduces Age-Related and Noise-Induced Hearing Loss

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High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness

(2013) Jennifer J Lentz et al.   NATURE MEDICINE

TMC1 and TMC2 Are Components of the Mechanotransduction Channel in Hair Cells of the Mammalian Inner Ear

(2013) Bifeng Pan et al.   NEURON

Genetics of Hearing and Deafness

(2012) Simon Angeli et al.   Anatomical Record-Advances in Integrative Anatomy and Evolutionary Biology

Gene transfer in inner ear cells: a challenging race

(2012) R Sacheli et al.   GENE THERAPY