The world stands on the edge of an era when gene editing can address many serious ills plaguing humankind, according to a pioneer of the revolutionary gene editing technique known as CRISPR-Cas9. But first, she said, there is a problem to solve.
Doudna, who visited
That process, while groundbreaking, is physically challenging for patients, and expensive, with each treatment costing more than
'It's exciting, but that's quite a small number,' Doudna said.
Doudna delivered her talk, 'Rewriting the Future of Health Care with Genome Editing,' on Thursday in a packed
She said that if CRISPR is to match its promise to reduce human suffering, new delivery methods are essential. She described several efforts underway in her lab and those of colleagues to create nanoparticle delivery systems that could, if perfected, relatively simply and cheaply deliver the CRISPR-based gene editor to target cells in various tissues.
That would allow the gene-editing process to occur inside the patient's body rather than in the lab, as occurs with the new sickle-cell treatment. That would avoid the expensive and arduous process of extracting cells from a patient's body, engineering them to address a condition's genetic causes, and then reinjecting them into the patient.
'How we can achieve in vivo genome editing, I increasingly think this is the bottleneck in this field,' Doudna said. 'Broadly speaking, what we need to be addressing is how these editors are going to get into target cells in the body. It's a really interesting, really big challenge, and there's many people working on it.'
The discovery of CRISPR/Cas9 in 2012 stemmed from basic scientific research into how bacteria fight off viruses. Researchers realized that a portion of the bacterial immune system contains molecules that precisely snip DNA at specific locations, and developed that into the molecular scissors of CRISPR/Cas9 that allow the precise editing of human, plant, and animal DNA at specific locations.
The technique was immediately seen as a major advance and other scientists began using it in their own research.
'Her groundbreaking development of CRISPR/Cas9 genome editing technology, with collaborator
Doudna, who received her Ph.D. from
'It makes me think that ultimately ... we can come up with a strategy for a particle that will be both easy to make, easy to program, and be effective at delivering in vivo,' Doudna said.
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