February 1, 2019
Scientists have long known what causes sickle-cell disease and its devastating effects: a single mutation in one errant gene. But for decades, there has been only modest progress against an inherited condition that mainly afflicts people of African descent.
With advances in gene therapy, that is quickly changing — so much so that scientists have begun to talk of a cure.
December 18, 2018
The Bill & Melinda Gates Foundation has donated $1.5 million to Boston Children’s Hospital to develop a gene therapy for sickle cell disease, with the goal of making the drug technology more widely available in regions of the world with high rates of the condition.
December 3, 2018
The potential therapies being developed and tested for sickle cell manipulate genes in a variety of ways, giving researchers a multitude of options and reason for hope.
May 23, 2018
The fifth installment in NOVA's 6-part series, "NOVA Wonders," which aired Wednesday, May 23rd,
features David Williams, MD, Boston Children's Chief Science Officer, Chief of the Division of Hematology and Oncology, and principal investigator on the trial, with Christine Duncan, MD, co-lead author on the study and a pediatric hematologist/oncologist at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and Colleen Dansereau, MSN, RN, CPN, CPHON, Program Manager for Boston Children's Gene Therapy Program.
December 7, 2017
MedicalXpress reports that a therapeutic technique to transplant blood-forming stem cells directly into the brain - a technique reported in Science Advances yesterday - could herald a revolution in our approach to treating central nervous system diseases and neurodegenerative disorders.
September 18, 2017
On Sunday, September 24, 2017, Hyundai Hope On Wheels® will present Dana-Farber Cancer Institute with a total of $400,000 from the Hyundai Scholar Hope and Young Investigator grant.
August 31, 2017
Dana-Farber/Boston Children's is actively preparing to offer the novel treatment.
January 19, 2017
A team of Dana-Farber scientists has released new research with an important message about precision medicine: Sequencing the genes of brain tumors in kids could point to treatments that target their genetic abnormalities and therefore have the best chance of being effective.
December 7, 2016
Colton was born healthy almost two years ago, but his fraternal twin brother, Levi, was not. The Michigan toddler had severe combined immune deficiency – “bubble boy” disease – discovered through newborn screening, Fox 17 reports from Grand Rapids.
November 4, 2016
Boston Children’s Hospital has established a new executive role to focus on research, the hospital announced.
Dr. David Williams, who serves as chief of hematology and oncology and director of clinical and translational research at Boston Children’s Hospital, will serve as chief scientific officer.