USH 1B
Dr. Alberto Auricchio from Naples (Italy) is doing a study into gene therapy for USH 1B. He is studying the possibility to deliver a healthy MYO7A gene by means of a double AAV vector in the retina.
USH 1B
In 2018, Sanofi started a clinical trial of gene therapy using a lentivirus and this trial has been stopped by now. Still, a second trial was started, which follows the long-term effects of those people who have participated in the terminated SAR421869 trial phase 1/2.
USH 1B
(Source: SaveSightNow: status and content of study are unknown)
Also Shannon Boye, Ph.D at the University of Florida, is engaged in the development of gene therapy using a double AAV vector for USH 1B.
USH 1C
Gwenaëlle Géléoc is assistant professor at the Boston Children’s Hospital and the Harvard Medical School. She did research into gene therapy for USH 1C.
USH 1D
(Source: Usher Coalition. Status and content unknown)
A research team in Naples, Italy, tested gene therapy with mice and pigs retinas with USH 1D using triple AAV vectors.
USH 1F
Livia Carvalho and the team of the University of West-Australia work together with Zubair Ahmed of the University of Maryland in the development of gene therapy for USH 1F. They have developed a double AAV vector that is being tested in a USH 1F mouse model.
USH 1F
Alex Hewitt and the team of Centre Eye Research Australia (CERA) are conducting a study for gene processing to find an effective treatment for the loss of eyesight associated with Usher 1F.
USH 1F
(Source: Usher Coalition. Status and content unknown)
Byrne and the team of the University of Pittsburgh are developing a ‘split gene dual vector system’ for gene therapy. This approach should increase the efficiency of viral vectors and so increase the gene expression.
USH 1F
(Source: Usher1F Collaborative)
Researchers at Harvard Medical School, Massachusetts Eye and Ear, and The Ohio State University, have created a mini-PCDH15 gene (USH1F) leading to an important step in developing gene therapy for USH1F. In testing the function of the mini-gene on deaf mice with USH1F, researchers recorded an increase in the production of the missing protein, enabling the hair cells to sense sound and restoring hearing.
USH 1G
Saaïd Safieddine and Christine Petit of the Institut Pasteur in France are busy developing gene therapy for the USH 1G gene. Using a mouse model, scientists have succeeded in recovering the hearing and the balance with one single local injection.
USH 2A
Erwin van Wijk is researcher at the Radboudumc Nijmegen, the Netherlands, and studies the question whether the functionality of various artificial, short forms of the USH 2A protein (coded by so-called “mini-genes”) is sufficient to inhibit or even stop the deterioration of the eyesight.
USH 2C
Erwin van Wijk is researcher at the Radboudumc Nijmegen, the Netherlands, and studies the question whether the functionality of various artificial, short forms of the USH 2C protein (coded by so-called “mini-genes”) is sufficient to inhibit or even stop the deterioration of the eyesight.
USH 2D
In a study of the group of Wade Chien (NIDCD, Bethesda, USA) a healthy copy of the whirlin gene was delivered using an AAV vector to the inner ear of new-born mouse models of which the whirlin gene had been disabled.
USH 3A
Dr. Kumar Alagramam of the University Hospital in Cleveland (Ohio, USA) is doing research into gene therapy with a focus on the development of a treatment for progressive loss of hearing with patients suffering from Usher Syndrome type 3A.
USH 3A
Erwin van Wijk is researcher at the Radboudumc Nijmegen, the Netherlands, and studies the question whether the functionality of various artificial, short forms of the USH 2C protein (coded by so-called “mini-genes”) is sufficient to inhibit or even stop the deterioration of the eyesight.
