Disease mechanisms and gene therapy for Usher syndrome

Geleoc, GGS; El-Amraoui, A

Geleoc, GGS (corresponding author), Boston Childrens Hosp, 3 Blackfan Circle,03001, Boston, MA 02115 USA.; Geleoc, GGS (corresponding author), Harvard Med Sch, Ctr Life Sci, 3 Blackfan Circle,03001, Boston, MA 02115 USA.; El-Amraoui, A (corresponding author), Sorbonne Univ, Inst Pasteur, Unit Progress Sensory Disorders, INSERM,UMRS1120, 25 Rue Dr Roux, F-75015 Paris, France.

HEARING RESEARCH, 2020; 394 ():

Abstract

Usher syndrome (USH) is a major cause of deaf-blindness in humans, affecting similar to 400 000 patients worldwide. Three clinical subtypes, USH1-3, h......

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