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David Williams, MD

Gene therapy halts progression of cerebral adrenoleukodystrophy in clinical trial

October 04, 2017
The devastating neurodegenerative disease typically affects young boys and causes death within 10 years
gene therapy Coopersville twins

Gene Therapy 2.0

March 15, 2017
Levi Looks, diagnosed through newborn screening with “bubble boy” disease, is a healthy toddler thanks to gene therapy, resurgent now after early setbacks, MIT Technology Review reports.
red blood cells

One boy’s cure raises hopes and questions about gene therapy for sickle cell disease

March 01, 2017
Dana-Farber/Boston Children’s researchers, who are developing a gene therapy trial for sickle cell disease, commented in Stat and other outlets on a case report from a different trial.
gene therapy Coopersville twins

Gene therapy trial gives Coopersville toddler second chance at life

December 07, 2016
A West Michigan toddler born with “bubble boy” disease is active and healthy, thanks to newborn screening and gene therapy, Fox 17 TV in Grand Rapids reports.
Alessandra Biffi

Pioneering doctor reflects on gene therapy’s trajectory

November 23, 2016
Alessandra Biffi, MD, director of gene therapy at Dana-Farber/Boston Children’s answers New Scientist’s questions about the resurgence of gene therapy after early setbacks.
Boy treated with Gene Therapy for MLD

Fighting genetic disease with help of HIV virus

October 23, 2016
A gene therapy trial in Italy led by Alessandra Biffi, MD, who now directs the Gene Therapy Program at Dana-Farber/Boston Children’s, has had promising results treating a devastating, lethal inherited neurological disorder, CBS Sunday Morning reports.
StuartOrkin_Alt

Gene therapy offers hope for treatment of sickle cell anemia

October 01, 2016
Scientists are finalizing plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anemia, the Guardian reports. The technique could begin trials next year, say researchers.
patient-and-doctor-CGD

Finally, hope for a young patient with CGD

September 14, 2016
At 16, Brenden was so sick that he started planning his funeral, Harvard Gazette reports. Today, Brenden, who has a rare immune disorder called chronic granulomatous disease, is 23 and preparing, with cautious optimism, to return to college. What changed? He participated in a gene therapy clinical trial to correct a disease that severely weakens the infection-fighting abilities.
Hemoglobin F - sickle cell

BCL11A-based gene therapy for sickle cell disease passes key preclinical test

September 06, 2016
A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, according to a study in the Journal of Clinical Investigation. The work sets the stage for bringing a decades-old discovery about sickle cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in 2017.
Alessandra Biffi

Gene therapy trial wrenches families as one child’s death saves another

July 20, 2016
MIT Technology Review reports on a gene therapy trial in Italy that has reported strong early results for an inherited neurodegenerative disease. The trial, led by Alessandra Biffi, MD – now director of gene therapy at Dana-Farber/Boston Children’s – helped a number of babies identified through newborn screening after an older sibling died of metachromatic leukodystrophy (MLD).
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