Health & Medicine
Genetic Diseases May Receive Help from Modified HIV Virus
Kathleen Lees
First Posted: Jul 12, 2013 12:11 AM EDT
Recent research shows promising results for children with hereditary diseases like metachromatic leukodystrophy and Wiskott Aldrich syndrome who undergo gene therapy vectors derived from the HIV virus.
"Three years after the start of the clinical trial, the results obtained from the first six patients are very encouraging: the therapy is not only safe, but also effective and able to change the clinical history of these severe diseases," lead researcher Luigi Naldini said in a news release.
According to the study, researchers believe that both leukodystrophy and Wiskott-Aldrich syndrome may be caused by a genetic defect that results in the deficiency of a protein essential for the organism in the early years of life.
Researchers looked to the therapy in order to determine if it could help alleviate problems affected by the disease.
The study started in 2010 and involved 16 participants, six of which suffered from Wiskott-Aldrich syndrome and 10 from metachromatic leukodystrophy.
However, the study just looked at the first six patients suffering from the syndrome. The researchers concluded the following, via a statement below from Alessandro Aiuti, coordinator of the clinical study on these patients and Head of Research of the Pediatric Clinic at Telethon Institute for Gene Therapy.
Aiuti adds that the gene therapy can help children who face problems with severe bleeding and infections lead a normal life.
"In the case of metachromatic leukodystrophy, however, the therapeutic mechanism is more sophisticated: the corrected hematopoietic cells reach the brain through the blood and release the correct protein that is 'gathered' there by the surrounding nerve cells. The winning card was to make engineered cells able to produce a quantity of protein much higher than normal, and thus effectively counteract the neurodegenerative process," researcher Alessandra Biffi said, according to a news release.
More information regarding the study can be found in the journal Science.
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First Posted: Jul 12, 2013 12:11 AM EDT
Recent research shows promising results for children with hereditary diseases like metachromatic leukodystrophy and Wiskott Aldrich syndrome who undergo gene therapy vectors derived from the HIV virus.
"Three years after the start of the clinical trial, the results obtained from the first six patients are very encouraging: the therapy is not only safe, but also effective and able to change the clinical history of these severe diseases," lead researcher Luigi Naldini said in a news release.
According to the study, researchers believe that both leukodystrophy and Wiskott-Aldrich syndrome may be caused by a genetic defect that results in the deficiency of a protein essential for the organism in the early years of life.
Researchers looked to the therapy in order to determine if it could help alleviate problems affected by the disease.
The study started in 2010 and involved 16 participants, six of which suffered from Wiskott-Aldrich syndrome and 10 from metachromatic leukodystrophy.
However, the study just looked at the first six patients suffering from the syndrome. The researchers concluded the following, via a statement below from Alessandro Aiuti, coordinator of the clinical study on these patients and Head of Research of the Pediatric Clinic at Telethon Institute for Gene Therapy.
Aiuti adds that the gene therapy can help children who face problems with severe bleeding and infections lead a normal life.
"In the case of metachromatic leukodystrophy, however, the therapeutic mechanism is more sophisticated: the corrected hematopoietic cells reach the brain through the blood and release the correct protein that is 'gathered' there by the surrounding nerve cells. The winning card was to make engineered cells able to produce a quantity of protein much higher than normal, and thus effectively counteract the neurodegenerative process," researcher Alessandra Biffi said, according to a news release.
More information regarding the study can be found in the journal Science.
See Now: NASA's Juno Spacecraft's Rendezvous With Jupiter's Mammoth Cyclone