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- Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
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Promising 'Mini Gene' Therapy for Usher Syndrome Type 1F
USH1F Current Research : USH Gene-Specific Research : Research : Usher Syndrome Coalition
The genetic and phenotypic landscapes of Usher syndrome: from disease mechanisms to a new classification
AAV-mini-PCDH15s rescue hearing in the Myo15-Cre mouse model of USH1F a
Case report: Compound heterozygous nonsense PCDH15 variant and a novel deep‐intronic variant in a Chinese child with profound hearing loss - Yang - 2023 - Molecular Genetics & Genomic Medicine - Wiley Online Library
WO2020219990A1 - Aav vectors encoding mini-pcdh15 and uses thereof - Google Patents
USH1F Current Research : USH Gene-Specific Research : Research : Usher Syndrome Coalition
Proposed therapy, developed in a Pcdh15-deficient mouse, for progressive loss of vision in human Usher syndrome
Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss: Molecular Therapy Methods & Clinical Development
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F: Molecular Therapy
Promising 'Mini Gene' Therapy for Usher Syndrome Type 1F
PDF] PCDH15 is expressed in the neurosensory epithelium of the eye and ear and mutant alleles are responsible for both USH1F and DFNB23.
