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International Gene Therapy Trial Launched at Children's Hospital Boston for "Bubble Boy" Syndrome

July 07, 2010
Safer delivery vehicles could advance gene therapy for many genetic diseases
David Williams, MD

Williams honored for Outstanding Achievement by ASGCT

January 23, 2012
Congratulations to David Williams, MD, chief of the Division of Hematology/Oncology and director of Translational Research at Children's, and associate chair of Pediatric Oncology at Dana-Farber Cancer Institute, who has been selected by the American Society of Gene & Cell Therapy as the recipient of their 2012 Outstanding Achievement Award.

Potential gene therapy approach to sickle cell disease highlighted at ASH

December 11, 2012
Proof-of-concept animal study selected as one of the best presentations at 54th American Society of Hematology Annual Meeting and Exposition

Gene therapy’s time seems to have come

July 14, 2013
Years after it was hyped, then vilified after the death of a teenager, then mostly forgotten by the general public, gene therapy has rebounded, and hospitals, companies, and investors in the Boston area have jumpedon the bandwagon. Patients are enrolling in a growing number of clinical trials here, and in some cases showing dramatic improvement.

Gene Therapy Trial for "Bubble Boy Syndrome"

August 23, 2013
Dana-Farber/Boston Children's David Williams, MD. is a guest on WGBH radio's innovation Hub to discuss a groundbreaking new treatment for Bubble Boy Syndrome.
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.
Dr. Sung-Yun Pai

International Gene Therapy Trial for ‘Bubble Boy’ Disease Shows Promising Early Results

December 07, 2013
(News release) Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as “bubble boy” disease, who were treated as part of an international clinical study of a new form of gene therapy.

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.
boy patient gene therapy trial

Gene Therapy Keeps “Bubble Boy” Disease at Bay in 8 Children

December 07, 2013
Researchers report promising results of a gene therapy trial aimed at curing the genetic defect that causes some children to be born without immune defenses, a rare condition also known as “bubble boy” disease, NPR reports.

‘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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