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‘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.
Alessandra Biffi

A new gene therapy transplantation technique could improve treatment of neurodegenerative disease

December 06, 2017
Alessandra Biffi, MD, led a study that shows the efficacy of a new gene therapy transplantation technique that could have future applications for treating neurodegenerative diseases.
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.
Research Test Tubes

Bluebird Bio Advances Experimental Drug for Genetic Brain Disorder

October 28, 2013
Bluebird bio Inc., a Cambridge gene therapy company, announced that the first patient has undergone infusion with its Lenti-D drug product in a stem cell transplantation designed to slow the progression of a genetic brain disorder called childhood cerebral adrenoleukodystrophy (CCALD), reports. The patient is being treated at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.
David Williams, MD

Boston Children’s Hospital to receive $1.5M to initiate basic work to expand gene therapy treatment of sickle cell disease into developing countries

December 19, 2018
The research will focus on adapting gene therapy for use in low-resource areas with high rates of the disease.

Discovery Holds Promise for 'Bubble Boy’ Patients

December 07, 2013
An international study led by Dana-Farber Cancer Institute and Boston Children's Hospital has shown that a new type of gene therapy may help boys with a fatal immune disorder commonly known as “bubble boy” disease, the Boston Business Journal reports.

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.
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.

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.
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.
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