Researchers Bring Gene Editing To Patients With Deadly Diseases
Until recently, gene therapy was laborious, crude and unsafe for human testing. However the new technology, called CRISPR-Cas9, works as a microscopic scalpel, performing genomic surgery with a accuracy, efficiency and affordability thought unimaginable. Porteus, an associate professor of pediatrics and a pioneer in gene editing. Meanwhile, researchers at Duke University and two other unbiased labs announced Jan. 1 they are using the same approach to fix a muscle gene, repairing function in mice with an incurable kind of muscular dystrophy. Their results were published in the journal Science. Boston research workers are deploying the tool to take care of a rare inherited eye disease that can cause blindness.
Other teams are working to repair the genes that cause Huntington’s disease, Sanfilippo symptoms and cystic fibrosis. CRISPR has been around the crosshairs of controversy due to its profound potential to rearrange the basic building blocks of life. But its healing promise is exactly what excites the medical community, especially as the price of the new technology gain access to and plunges expands. These older approaches could not guarantee that the new gene was spliced into the right place.
It also risked disruption of adjacent genes. While there have been recent improvements with two more precise techniques, they are time-consuming and tricky. CRISPR – which stands for “clustered regularly interspaced short palindromic repeats,” or clusters of brief DNA sequences that read likewise forwards and backward – is the overall game changer.
Only 3 years old, it works like the search-and-replace function of some type of computer. A Palo Alto indigenous who was informed at Stanford and Harvard, Porteus, 51, has spent his life time viewing helplessly as patients declined from blood and disease fighting capability diseases such as sickle cell anemia, beta thalassemia and severe mixed immunodeficiency disease. These are extremely different diseases, he said.
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But they have this in common: Each is caused by a single mutation in a single gene of a single cell type. A lot more than 10,000 human being diseases are caused by a one gene defect, regarding to Dr. Maria Grazia Roncarolo, co-director of Stanford’s Institute for Stem Cell Biology and Regenerative Medicine.
Porteus’ lab excises the bad gene, using CRISPR. Then his team introduces a good gene. The scientists intend to harvest some of the sick cells that provide rise to the blood or disease fighting capability cells, called stem cells, from the bone marrow of an individual. Then the patient would go through extensive chemotherapy to eliminate the remaining unwell stem cells and make space for new ones. Following the faulty genes in the harvested sick cells are fixed using CRISPR, the repaired cells would incubate for several days. Finally, they might infuse the corrected stem cells into the patient. Once in place, these stem cells would produce healthy cells to displace the ill cells – so the patient heals.