Now, personalized gene therapies for vision loss

A new approach to develop personalized gene therapies for people suffering from retinitis pigmentosa (RP) has been developed by the researchers.

 The RP is one of the major causes of vision loss.
The approach uses induced pluripotent stem cell technology to transform skin cells into retinal cells. These cells are then used by the researchers as a patient-specific model for disease study and preclinical testing.
The researchers from Columbia University Medical Center (CUMC) used this approach to show that a form of RP caused by mutations to the gene MFRP (membrane frizzled-related protein) can disrupt the protein that gives retinal cells their structural integrity.
"The use of patient-specific cell lines for testing the efficacy of gene therapy to precisely correct a patient's genetic deficiency provides yet another tool for advancing the field of personalised medicine," said Stephen H Tsang, the Laszlo Z Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology.
The RP could also begin during infancy but its first symptoms typically emerge in early adulthood, causing night blindness. In later stages, the photoreceptors in the macula are destroyed by the RP. The photoreceptors are responsible for fine central vision.
"This study provides both in vitro and in vivo evidence that vision loss caused by MFRP mutations could potentially be treated through AAV gene therapy," said coauthor Dieter Egli, an assistant professor at CUMC.
The paper was published in Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.

 

 

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