New revolutionary technology allows experts to rewrite the genetic code of patients giving hope for novel gene therapy. Genetic mutations that cause debilitating hereditary diseases, such as kidney disease, have been affecting children and young adults across the globe. Recently experts have successfully fixed patient-derived kidney cells using a novel DNA repair kit.
DNA Repair Kit: A New Hope for Hereditary Diseases
A recent study published in the journal Nucleic Acids Research, titled "Highly efficient CRISPR-mediated large DNA docking and multiplexed prime editing using a single baculovirus." An international team of researchers led by experts from the University of Bristol has developed a DNA repair vehicle that can genetically fix flawed podocin, a usual cause of inheritable Steroid Resistant Nephrotic Syndrome.
Podocin, a protein, is located on the surface of specialized kidney cells vital for kidney functions. On the other hand, faulty podocin remains stuck inside the kidney cells and never makes it to the organ's surface, causing terminal damage to the podocytes. Since the disease cannot be cured with any medication, gene therapy repairs offer hope to patients by repairing the genetic mutation at its roots, says EurekAlert.
Conventionally, experts have used human viruses in gene therapy applications to carry genetic repairs. These are used like Trojan Horses to enter cells that carry the genetic errors. As of now, lentivirus, adenovirus, and adeno-associated viruses, harmless viruses that infect humans, dominate the system.
However, the commonly used viruses share the same limitations regarding being restricted to the space within the virus's viral shells. In turn, this constrains the cargo these viruses can deliver, namely the DNA kit needed for the efficient repair of genetics, significantly limiting the scope of the virus's application for gene therapy.
Re-Engineering Baculovirus for Efficient DNA Repair-Kit Transport
The team led by Bristol's School of Biochemistry, Dr. Francesco Aulicino, and Professor Imre Berger applied synthetic biology techniques. It re-engineered the baculovirus, a harmless human insect virus, to no longer be constrained by limiting cargo capacity.
Auclino, who led the study, says that what sets the baculovirus apart from other viruses commonly used for gene therapy is the lack of a rigid shell encapsulating the cargo space. The shells of baculovirus are like hollow sticks; it becomes longer as more cargo is placed. This means that more sophisticated tool-kits to repair genetic defects can be efficiently delivered by the virus making it more versatile than the virus commonly used in the system.
First, the team had to enable baculovirus to penetrate human cells, which it doesn't normally do. Auclino and his team decorated the virus with proteins, allowing it to enter human cells efficiently. This modification still makes the virus safe, especially since it only multiplies in insects and not in human cells. The team then used the engineered baculovirus to deliver larger DNA pieces and build them into the genomes of a range of human cells.
Professor Gavin Welsh, a co-author of the study and a professor of Renal Cell Biology at the university, concluded that the study results are encouraging. Stating that the novel approach pioneered by Berger holds promise not only for SERNS patients but for a wide range of genetic diseases.
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