4.4 Article

AAV8BP2 and AAV8 transduce the mammalian cochlear lateral wall and endolymphatic sac with high efficiency

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Multidisciplinary Sciences

Gene therapy via canalostomy approach preserves auditory and vestibular functions in a mouse model of Jervell and Lange-Nielsen syndrome type 2

Xuewen Wu et al.

Summary: Gene therapy via canalostomy approach in mouse model of JLNS2 effectively preserved auditory and vestibular functions by early restoration of inner ear morphology, prevention of degeneration of inner ear cells, and dose-dependent treatment efficacy. Highly efficient transduction is crucial for maintaining long-term therapeutic effects, and the delivery method holds promise for human inner ear gene therapy.

NATURE COMMUNICATIONS (2021)

Article Multidisciplinary Sciences

Dissection of the Endolymphatic Sac from Mice

Keiji Honda et al.

Summary: Research on mutant mouse models of human hearing and balance disorders has unveiled changes in the development and function of neurosensory epithelia, but limited knowledge exists about the endolymphatic sac. Difficulty in visualizing and microdissecting this tissue, largely due to its single-cell layer composition, hinders further understanding of this understudied part of the inner ear.

JOVE-JOURNAL OF VISUALIZED EXPERIMENTS (2021)

Article Neurosciences

Single-Cell RNA-Seq of Cisplatin-Treated Adult Stria Vascularis Identifies Cell Type-Specific Regulatory Networks and Novel Therapeutic Gene Targets

Ian A. Taukulis et al.

Summary: This study established a model of EP dysfunction by administering a single dose of cisplatin to adult mice, identifying transcriptional changes induced by cisplatin exposure and accompanying EP reduction. It also identified gene regulatory networks unique to cisplatin-treated SV and potential druggable gene targets within those networks, providing insights for interventions in cisplatin ototoxicity and EP dysfunction.

FRONTIERS IN MOLECULAR NEUROSCIENCE (2021)

Review Audiology & Speech-Language Pathology

Genome and base editing for genetic hearing loss

Philipp Niggemann et al.

HEARING RESEARCH (2020)

Review Audiology & Speech-Language Pathology

Adeno-associated virus gene replacement for recessive inner ear dysfunction: Progress and challenges

Charles Askew et al.

HEARING RESEARCH (2020)

Article Multidisciplinary Sciences

Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

Omar Akil et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

Article Multidisciplinary Sciences

AAV2.7m8 is a powerful viral vector for inner ear gene therapy

Kevin Isgrig et al.

NATURE COMMUNICATIONS (2019)

Article Medicine, Research & Experimental

A dual-AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock-out mice

Hanan Al-Moyed et al.

EMBO MOLECULAR MEDICINE (2019)

Article Multidisciplinary Sciences

Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders

Carl A. Nist-Lund et al.

NATURE COMMUNICATIONS (2019)

Article Medicine, Research & Experimental

Clarin-1 gene transfer rescues auditory synaptopathy in model of Usher syndrome

Didier Dulon et al.

JOURNAL OF CLINICAL INVESTIGATION (2018)

Review Biotechnology & Applied Microbiology

Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina

Pavitra S. Ramachandran et al.

HUMAN GENE THERAPY (2017)

Article Biotechnology & Applied Microbiology

Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome

Kevin Isgrig et al.

MOLECULAR THERAPY (2017)

Article Biotechnology & Applied Microbiology

Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV

Bence Gyorgy et al.

MOLECULAR THERAPY (2017)

Article Biotechnology & Applied Microbiology

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

Bifeng Pan et al.

NATURE BIOTECHNOLOGY (2017)

Article Multidisciplinary Sciences

Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G

Alice Emptoz et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

Article Multidisciplinary Sciences

In vivo genetic manipulation of inner ear connexin expression by bovine adeno-associated viral vectors

Giulia Crispino et al.

SCIENTIFIC REPORTS (2017)

Review Neurosciences

DFNB1 Non-syndromic Hearing Impairment: Diversity of Mutations and Associated Phenotypes

Francisco J. del Castillo et al.

FRONTIERS IN MOLECULAR NEUROSCIENCE (2017)

Article Multidisciplinary Sciences

Modeling and Preventing Progressive Hearing Loss in Usher Syndrome III

Ruishuang Geng et al.

SCIENTIFIC REPORTS (2017)

Review Oncology

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

Michael F. Naso et al.

BIODRUGS (2017)

Article Genetics & Heredity

Comprehensive genetic testing in the clinical evaluation of 1119 patients with hearing loss

Christina M. Sloan-Heggen et al.

HUMAN GENETICS (2016)

Article Biotechnology & Applied Microbiology

Gene Therapy Restores Hair Cell Stereocilia Morphology in Inner Ears of Deaf Whirler Mice

Wade W. Chien et al.

MOLECULAR THERAPY (2016)

Article Cell Biology

Tmc gene therapy restores auditory function in deaf mice

Charles Askew et al.

SCIENCE TRANSLATIONAL MEDICINE (2015)

Review Audiology & Speech-Language Pathology

The enigmatic root cell - Emerging roles contributing to fluid homeostasis within the cochlear outer sulcus

Daniel J. Jagger et al.

HEARING RESEARCH (2013)

Article Neurosciences

The Membrane Properties of Cochlear Root Cells are Consistent with Roles in Potassium Recirculation and Spatial Buffering

Daniel J. Jagger et al.

JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY (2010)

Review Medicine, General & Internal

Current concepts: Newborn hearing screening - A silent revolution

CC Morton et al.

NEW ENGLAND JOURNAL OF MEDICINE (2006)

Article Biotechnology & Applied Microbiology

Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fluorescent proteins

RL Klein et al.

MOLECULAR THERAPY (2006)

Article Medicine, General & Internal

Presbycusis

GA Gates et al.

LANCET (2005)

Review Audiology & Speech-Language Pathology

K+ cycling and the endocochlear potential

P Wangemann

HEARING RESEARCH (2002)