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

被引：0
作者：
Bifeng Pan
Charles Askew
Alice Galvin
Selena Heman-Ackah
Yukako Asai
Artur A Indzhykulian
Francine M Jodelka
Michelle L Hastings
Jennifer J Lentz
Luk H Vandenberghe
Jeffrey R Holt
Gwenaëlle S Géléoc
机构：
[1] F.M. Kirby Center for Neurobiology,Department of Otolaryngology
[2] Boston Children's Hospital,Department of Neurobiology
[3] Harvard Medical School,Department of Cell Biology & Anatomy
[4] Harvard Medical School,Department of Otorhinolaryngology & Bio
[5] Chicago Medical School,communications and Neuroscience Center
[6] Rosalind Franklin University of Medicine and Science,Department of Ophthalmology
[7] LSU Health Sciences Center,Department of Neurology
[8] Grousbeck Gene Therapy Center,undefined
[9] Schepens Eye Research Institute and Massachusetts Eye and Ear,undefined
[10] Harvard Medical School,undefined
[11] F.M. Kirby Center for Neurobiology,undefined
[12] Boston Children's Hospital,undefined
[13] Harvard Medical School,undefined
[14] Present addresses: Gene Therapy Center,undefined
[15] University of North Carolina,undefined
[16] Chapel Hill,undefined
[17] North Carolina,undefined
[18] USA (C.A.); MedStar Washington Hospital Center,undefined
[19] Georgetown University Medical Center,undefined
[20] Washington,undefined
[21] DC,undefined
[22] USA (S.H.-A.).,undefined
来源：
Nature Biotechnology | 2017年 / 35卷
关键词：
D O I：
暂无
中图分类号：
学科分类号：
摘要：
Gene therapy in the inner ear achieves unprecedented recovery of hearing and balance behavior in Usher syndrome mice.
引用
收藏
页码：264 / 272
页数：8
相关论文
共 50 条
  • [41] Aldh inhibitor restores auditory function in a mouse model of human deafness
    Zhu, Guang-Jie
    Gong, Sihao
    Ma, Deng-Bin
    Tao, Tao
    He, Wei-Qi
    Zhang, Linqing
    Wang, Fang
    Qian, Xiao-Yun
    Zhou, Han
    Fan, Chi
    Wang, Pei
    Chen, Xin
    Zhao, Wei
    Sun, Jie
    Chen, Huaqun
    Wang, Ye
    Gao, Xiang
    Zuo, Jian
    Zhu, Min-Sheng
    Gao, Xia
    Wan, Guoqiang
    [J]. PLOS GENETICS, 2020, 16 (09): : e1009040
  • [42] A gene for recessive nonsyndromic sensorineural hearing impairment (DFNB18) maps to the chromosomal region containing the Usher Syndrome type 1C gene.
    Jain, PK
    Lalwani, AK
    Li, XC
    McDuffie, T
    Deshmukh, D
    Verma, IC
    Wilcox, ER
    [J]. AMERICAN JOURNAL OF HUMAN GENETICS, 1997, 61 (04) : A280 - A280
  • [43] Cochlear gene therapy restores hearing and auditory processing in an atypical DFNB9 mouse model
    Najate Benamer
    Hélène Le Ribeuz
    Chloé Felgerolle
    Charlotte Calvet
    Olivier Postal
    Baptiste Plion
    Mauricio Saenz-Roldan
    Marie Giorgi
    Marie-José Lecomte
    Yann Nguyen
    Christine Petit
    Nicolas Michalski
    Boris Gourévitch
    Omar Akil
    Saaid Safieddine
    [J]. Communications Medicine, 5 (1):
  • [44] A CHROMOSOME 11P14-15.1 YAC CONTIG THAT ENCOMPASSES THE USHER SYNDROME TYPE 1C LOCUS
    AYYAGARI, R
    NESTOROWICZ, A
    LI, Y
    SMITH, RJH
    KAISERKUPFER, M
    PERMUTT, MA
    HEJTMANCIK, JF
    [J]. AMERICAN JOURNAL OF HUMAN GENETICS, 1995, 57 (04) : 1484 - 1484
  • [45] AAV Mediated Gene Therapy Restores Partial Auditory Sensitivity in Mice Models of Autosomal Recessive Non Syndromic Deafness DFNB31 and Usher Syndrome Type IID
    Goldberg, Hannah
    [J]. MOLECULAR THERAPY, 2020, 28 (04) : 144 - 144
  • [46] Gene Therapy Restores Normal Weight in a Obese Bardet-Biedl Syndrome 1 (BBS1) Mouse Model
    Jeyabalan, Jeshmi
    Munye, Mustafa
    Castro, Sandra
    Chawda, Mira
    Rahim, Ahad
    Waddington, Simon
    Beales, Phillip L.
    Hernandez-Hernandez, Victor
    [J]. MOLECULAR THERAPY, 2018, 26 (05) : 255 - 256
  • [47] Correction of visual and auditory function by AAV9-mediated gene therapy in a mouse model of mucopolysaccharidosis type IIIB
    Ribera, A.
    Motas, S.
    Ramirez, L.
    Marco, S.
    Sanchez, V.
    Sanchez, X.
    Bertolin, J.
    Perez, J. A.
    Leon, X.
    Varela-Nieto, I.
    de la Villa, P.
    Haurigot, V.
    Bosch, F.
    [J]. HUMAN GENE THERAPY, 2017, 28 (12) : A67 - A67
  • [48] A Natural History Study of Visual Function in Patients with Usher Syndrome Type 1C.
    Hartman, Meredith Harrell
    Robillard, Katelyn N.
    Sun, Vincent
    Lopez, Irma
    Ibrahim, Mariam
    Walsdorf, Kelsey
    Reinoso, Maria
    Koenekoop, Robert K.
    Lentz, Jennifer J.
    [J]. INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2018, 59 (09)
  • [49] Correction of Visual and Auditory Function by AAV9-Mediated Gene Therapy in a Mouse Model of Mucopolysaccharidosis Type IIIB
    Ribera, Albert
    Motas, Sandra
    Ramirez, Laura
    Marco, Sara
    Sanchez, Victor
    Sanchez, Xavier
    Bertolin, Joan
    Perez, Jennifer A.
    Leon, Xavier
    Varela-Nieto, Isablel
    de la Villa, Pedro
    Haurigot, Virginia
    Bosch, Fatima
    [J]. MOLECULAR THERAPY, 2018, 26 (05) : 114 - 114
  • [50] Nonhuman primate model of Usher Syndrome Type 1B: course of retinal degeneration and initial gene therapy results
    Renner, Lauren
    Ryu, Junghyun
    Hennebold, Jon D.
    Boye, Sanford L.
    Hanna, Carol
    Burch, Fernanda
    Kempton, Beth
    Porsov, Edward V.
    Spears, Rachel
    Burwitz, Benjamin
    Lauer, Andreas K.
    Wilson, David
    Pennesi, Mark E.
    Boye, Shannon
    Brigande, John V.
    Neuringer, Martha
    [J]. INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2023, 64 (08)
12345