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#GeneEditing

Positive Reads

Breakthrough Enzyme Enhances Precision of CRISPR Gene Editing Technology

In a major scientific advancement, researchers at Duke University have developed a groundbreaking enzyme that significantly expands the capabilities of CRISPR gene editing technology. This breakthrough allows for precise targeting of almost any human gene, opening up new possibilities for treating a wide range of diseases.   The original CRISPR system could only target a limited portion of the human genome, limiting its potential applications. However, the new method developed by the Duke University engineers overcomes this limitation, providing access to nearly every gene in the human genome. This advancement has the potential to revolutionize the field of gene therapy and genome engineering. Collaborating with renowned institutions such as Harvard University, Massachusetts Institute of Technology, University of Massachusetts Medical School, University of Zurich, and McMaster University, the Duke University team published their findings in the prestigious journal Nature Communications. By utilizing the CRISPR-Cas system, which is an immune system present in bacteria, the researchers were able to employ RNA molecules and CRISPR-associated (Cas) proteins to accurately target and destroy the DNA of specific genes. This precision allows for the modification of previously inaccessible genetic mutations associated with diseases such as Rett syndrome and Huntington's disease. The new enzyme, named SpRYc, combines the strengths of two separate variants, offering improved accuracy and a broader range of DNA editing possibilities. While further research is needed to explore its full potential, the development of this enzyme marks a significant step forward in the field of gene editing. With the ability to target almost any human gene with precision, scientists are now poised to unlock new treatment options and potentially find cures for previously untreatable genetic disorders. The future of precision medicine looks brighter than ever with the advent of this groundbreaking enzyme.

7 Jan 2024
Positive Reads

Good News

A New Era in Medicine: FDA Grants Approval for CRISPR in Sickle Cell Treatment

The US Food and Drug Administration (FDA) has granted approval for the first-ever treatment using the gene-editing tool CRISPR to cure sickle cell disease, bringing renewed hope to thousands of patients. The treatment, known as Casgevy and manufactured by Vertex Pharmaceuticals, marks a significant milestone in the field of medical breakthroughs. Sickle cell disease affects over 100,000 Americans, causing severe pain and a shortened lifespan. Until now, the only available treatment option was a risky bone marrow transplant, which often faced challenges due to finding a suitable donor. However, the CRISPR technology offers a promising alternative by editing the genetic code of the patient's own stem cells. "This approval is a remarkable achievement, considering the rapid progress from the discovery of CRISPR to its recognition with a Nobel Prize and now as an approved product," said Dr. Alexis Thompson, chief of the division of hematology at Children's Hospital of Philadelphia. While the process involves multiple appointments and can be painful, patients like LaRae Morning, a 29-year-old recipient of the treatment, are experiencing life-changing benefits. Morning expresses gratitude for the newfound ability to engage in physical activities that were previously impossible. However, the treatment does come with a high price tag, costing $2.2 million. Despite the cost, insurance coverage may alleviate some financial burdens, considering the potential long-term savings in medical care. The FDA's approval of this groundbreaking CRISPR treatment for sickle cell disease ignites hope for patients and paves the way for further advancements in gene-editing technology. It opens up possibilities for addressing other genetic conditions and brings us one step closer to a future where genetic diseases can be treated effectively.

20 Dec 2023
Good News

Global Inspirations

In A First, Scientists Edit DNA In Attempt To Cure Genetic Disease

Scientists for the first time have edited the DNA of a patient in a bold attempt to cure a genetic disease. On Monday, 44-year-old Brian Madeux from California received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot. Madeux who has a metabolic disease called the Hunter syndrome said he was humbled to take the test. He added that he was willing to take the risk and hoped that it would help him and other people as well.  The result of the test would be known only after a month and can be confirmed only after three months. If it is a success, the new technique could give a major boost to the fledgling field of gene therapy. Scientists have edited genes before, but by altering cells in labs and then returning them to patients. In the new technique, genetic tinkering is happening in a precise way inside the body – like sending a miniature surgeon along to place the new gene in exactly the right location. Dr Sandy Macrae, president of Sangamo Therapeutics said, “We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending.It becomes part of your DNA and is there for the rest of your life.” The success of this therapy will help people who are suffering from incurable metabolic diseases.    

17 Nov 2017
Global Inspirations

Sickle Cell Disease is “Cured” first time in the World.

A considerable number of people around the world suffer from anemia. The anemia can be caused by various reasons. One of them is Sickle Cell Disease anemia, which was considered incurable. But, the scientists have been able to cure a teenager patient for the first time in the world, according to the reports in media. The researchers have treated a teenager’s sickle cell anemia by new gene-editing treatment successfully. In Sickle Cell Disease, defective blood cells are created by body’s bone marrow, which are in the shape of a sickle. Normally, cells are in a shape of sphere. What is more, the sickle-shaped cells cause blockage of  blood flow throughout the body. It results in extreme pain to the patient. Sickle cell disease affects people around the world to a large extend. The patient who received the break through therapy is said to have no disease-inflicted damage on his internal organs which needed blood transfusion while on medication every month. The research was conducted by the doctors at the Necker Children’s Hospital in Paris. They first drained out the bone marrow of the teenage patient.  Then the marrow was “infected” with a virus containing genetic instructions for manufacturing healthy red blood cells, in place of incorrect cells which blocked flow of blood. When this modified bone marrow was infused into the teenager body more than a year ago, he was able to discontinue medication. Further, he has not shown any symptoms of sickle cell disease after the treatment. Though the test was successful, the doctors have not called it a “cure” as some more tests are required to be conducted. Still, the achievement has given hope for millions of patients suffering from the Sickle Cell Disease anemia around the world.  

27 Mar 2017
Global Inspirations

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