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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).
StuartOrkin

Science commentary explores ways to pay for success in gene therapy

May 26, 2016
Noting the potential of gene therapy to be a one-time treatment for rare and serious diseases that otherwise cost hundreds of thousands, if not millions, of dollars in chronic care over a lifetime, the authors suggest several models for valuing, pricing and developing gene therapy.
Sickle Cell Photo

We’ve known for 50 years what causes sickle cell disease. Where’s the cure?

May 19, 2016
Boston Globe Media’s Stat examines efforts to use gene therapy or gene editing – or develop a pill – to harness the latest research on sickle cell disease. “This is the right time for a sickle cell moonshot,” says Dana-Farber/Boston Children’s President David Williams, MD.
Sun-YunPai

Early Gene Therapy Results in Wiskott-Aldrich Syndrome Promising

December 06, 2015
Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology.
Gene Therapy: Cell Manipulation Laboratory

Gene Therapy for “Bubble Boy” Disease Appears to be Effective and Safe

October 09, 2014
A new form of gene therapy for boys with “bubble boy” disease appears to be not only effective but also may avoid the late-developing leukemia seen in a quarter of SCID-X1 patients in pioneering gene therapy trials in Europe more than a decade ago.
DavidDWilliams_Blue

‘Bubble Boy’ Disease, Nearly Always Fatal, Could Have a Cure

December 12, 2013
An international consortium of scientists, including researchers at Dana-Farber/Boston Children's, is finding promising early results in a new gene therapy clinical trial they say may lead to an eventual long-term cure for SCID-X1, ABC News reports.
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