CRISPR An Advancement in Gene Therapy

Release Date: 20-Sep-2019

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a gene-editing technique that enables the targeting and editing of specific sequences in the human DNA. It is available from a period about 2 years, and recently, researchers in the U.S. have started using it to alter the genes of adults suffering from progressive disorders and diseases.


In year 2018, a Chinese scientist He Jiankui performs a controversial experiment on twin babies. He used CRISPR to alter the genes of twin embryos whose father was HIV-positive. He wanted to trigger a mutation in the CCR5 gene, which, if successful, would reduce the girls immune to HIV. It is very risky experiment as CRISPR is associated unwanted gene edition.


Dublin-based Allergan   and Editas Medicine, which is a discovery-phase pharmaceutical company in Cambridge, Mass., are trying to perform experiment to observe the benefits of CRISPR in treatment of disorders. In July 2019, they announced that they were ready to enroll subjects in first-of-its-kind CRISPR-based therapy trials in Boston. They planned to generate a development that would involve gene editing inside the human body.


They used CRISPR in the treatment of Blindness. An EDIT-101 injection is administered to the eye, which delivers the gene-altering machinery directly to photoreceptor cells. It is injected behind the retina, which cuts out the mutated DNA, including the CEP290 gene responsible for progressive photoreceptor-cell loss, which leads to an inherited type of blindness.


Gene therapy involving the viral vectoris also gets success in curing eye-related diseases. Spark Therapeutics in Philadelphia has developed a similar gene therapy named Luxturna, which is used for patients with inherited retinal disease. It is almost similar to the worl of Allergan and Editas except the targeting gene. It is targeting the other gene named RPE 65.


Some other companies are also performing research works using CRISPR to treat different genetic effects. Zolgensma is another CRISPR based product by Chicago-based AveXis, is able to treats often-fatal spinal muscular dystrophy by injecting a fully functional copy of the human SMN gene into target motor neuron cells.


Kymraih by Novartis is another cell-based gene therapy that aims to cure acute lymphoblastic leukemia, a cancer of the bone marrow and blood. Two other CRISPR companies are Intellia Therapeutics of Cambridge and CRISPR Therapeutics of Zug, Switzerland. 

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