CRISPR Fix Blindness Causing Defective Gene That Lead To Retinitis Pigmentosa

Here’s another awesome research. A new gene editing technology called CRISPR fix blindness causing defective gene, yes it is used to successfully repair a genetic mutation that leads to Retinitis Pigmentosa. The research was conducted by Columbia University Medical Center (CUMC) and University of Iowa scientists.

CRISPR fix blindness causing defective gene

 

The study published just now marks the first time researchers have replaced a defective gene associated with a sensory disease in stem cells that were derived from a patient’s tissue.

Stephen Tsang, MD, PhD, the László Z. Bitó Associate Professor of Ophthalmology and associate professor of Pathology & Cell Biology at CUMC, said

Our vision is to develop a personalized approach to treating eye disease

The study’s senior author also said,

We still have some way to go, but we believe that the first therapeutic use of CRISPR will be to treat an eye disease. Here we have demonstrated that the initial steps are feasible.

The researchers took a sample of skin from a patient dealing with Retinitis Pigmentosa and created stem cells. CRISPR fix by repairing the defective gene, since the patient-derived stem cells still harbored the disease-causing mutation. These stem cells can potentially transformed into healthy retinal cells to be transplanted back into the same patient to treat vision loss.

Tsang also stated,

The X-linked form of retinitis pigmentosa is an ideal candidate for a precision medicine approach because a common mutation accounts for 90% cases.

Doctors can re-transplant the cells with slight fear of non-acceptance by the immune system, since the corrections are done in cells derived from the patient’s own tissue,

Tsang and the team also believe that the first clinical use of CRISPR could be for treating an eye disease because  the eye is easy to access for surgery, readily accepts new tissue, and can be noninvasively monitored. And these things would not be easier with other body parts. We’re hoping for more breakthrough in CRISPR fix for blindness.

The paper is titled, “Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells“.

Other contributors: Andrew Zheng and Yao Li from CUMC, and Alexander G. Bassuk and Vinit B. Mahajan from Iowa University.

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