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article imageNovel gene editing tool for blood disorders

By Tim Sandle     Nov 2, 2016 in Science
A novel gene-editing system, based on nanotechnology, could cure a genetic blood disorder. Research so far has been based on studies in mice. The non-invasive tool has been heralded a success.
The new method is based on peptide nucleic acid molecules, derived from a synthetic nucleotide. Nucleotides are organic molecules that serve as the subunits of nucleic acids like DNA (deoxyribonucleic acid) and RNA (ribonucleic acid). These were rendered from biocompatible nanoparticles.
The new process works by opening up DNA to allow binding near the target site. In studies conducted using mice, the peptide nucleic acid molecules, together with nanoparticles, were used to target a malfunctioning gene in order to address a blood disorder.
The blood disorder was beta thalassemia. Thalassaemia is the name for a group of inherited conditions that affect haemoglobin. The result is that people with the condition produce either no or too little haemoglobin. Haemoglobin is used by red blood cells to carry oxygen around the body. Those with the condition are typically anaemic, which leads to them being tired, short of breath and pale.
The new process is non-invasive and involves delivering the gene editing cocktail directly into mice (and, if eventually demonstrated as effective with people, the same delivery mechanism would be used). Genome editing is a type of genetic engineering whereby DNA is inserted, deleted or replaced in the genome of a living organism using "molecular scissors" (nucleases).
The mice treated with the peptide nucleic acid molecules showed a beneficial response, most notably elevated levels of haemoglobin. This continued for several months post-treatment. Gene editing in hematopoietic stem cells (the stem cells that give rise to all the other blood cells through the process of haematopoiesis) was also observed.
The new process was developed by scientists at Carnegie Mellon University. In a research note, the lead scientist, a Dr. Mark Saltzman comments: “The nanoparticles are a critical component of this gene editing technology. It was developed by, a collaborator on this project.”
The researcher explains a further benefit with the technology: “These nanoparticles are nontoxic and nonimmunogenic, already approved by FDA for treatment of neurodegenerative diseases.”
The research has been published in the journal Nature Communications. The research is titled “In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery.”
More about Gene editing, Blood disorder, Blood disease
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