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Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F

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Various therapeutic approaches are used for the Usher syndrome. (A)

Gene Therapy for Usher Syndrome Type 1F, David Corey, PhD

WO2020219990A1 - Aav vectors encoding mini-pcdh15 and uses thereof - Google Patents

Proposed therapy, developed in a Pcdh15-deficient mouse, for progressive loss of vision in human Usher syndrome

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

Promising 'Mini Gene' Therapy for Usher Syndrome Type 1F

The genetic and phenotypic landscapes of Usher syndrome: from disease mechanisms to a new classification

Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss: Molecular Therapy Methods & Clinical Development

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