When baby Layla's prognosis turned from bleak to hopeful following a successful application of genetic therapy to treat her leukemia, researchers became optimistic that gene editing can also be used to treat other diseases.
Layla Richards, who was diagnosed with acute lymphoblastic leukemia (ALL), is the first patient to be treated by gene editing performed by doctors from the Great Ormond Street Hospital in collaboration with biotech firm Cellectis.
While it is not exactly a cure, the genetic therapy technique was able to successfully and vastly improve the patient's condition.
"I didn't want to go down that road [and admit her to palliative care], I'd rather that she tried something new and I took the gamble," said Layla's father Ashleigh, who is incredibly grateful for the opportunity that may guarantee his daughter's continued survival.
The technique used in Layla's treatment was the transcription activator-like effector nuclease (TALEN) technology. Dubbed as the tiny scissors for genes, TALENs are enzymes that allow for the cutting of specific sequences in DNA strands and fixing broken genome sequences.
Technically, the use of TALENs has not been declared ready to be tested on people yet, which is why it was a huge gamble when Layla's parents and doctors opted to make use of it. But with the positive effect of the treatment on Layla, scientists hope that the TALEN technique could be the key to curing many other diseases.
"Our approach actually repairs the faulty gene, which is a lot simpler," explained David Ousterout, a biomedical engineering graduate student from Duke University who led the study on the use of TALENs for therapeutic use. "It finds the faulty gene, and fixes it so it can start producing a functional protein again."
Editing genes is no easy task since it can be detrimental to cells if used too forcefully, or may not have any effect if used too lightly. However, once the technique is perfected, scientists hope to be able to use it to cure hereditary disorders like sickle cell anemia, a blood disorder that causes deformed red blood cell formation; and cystic fibrosis, a disease that causes a buildup of thick mucus and damage in the lungs and digestive system due to a defective gene.
TALENs and other genome editing techniques may seem like the answer to managing genetic-related disorders, though experts remind the public that more research is needed to be done and that the therapy may not be for everyone.
"We have to be cautious about claiming that this will be a suitable treatment option for all children," noted Waseem Qasim, a gene therapy researcher from University College London. Qasim calls the treatment a "landmark" in the use of technology for genetic engineering. If replicated, the gene editing technique "could represent a huge step forward in treating leukemia and other cancers."
